<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT02999854</url>
  </required_header>
  <id_info>
    <org_study_id>CR-AIR-009</org_study_id>
    <secondary_id>2016-004672-21</secondary_id>
    <nct_id>NCT02999854</nct_id>
  </id_info>
  <brief_title>Safety and Efficacy of ATIR101 as Adjunctive Treatment to Blood Stem Cell Transplantation From a Haploidentical Family Donor Compared to Post-transplant Cyclophosphamide in Patients With Blood Cancer</brief_title>
  <acronym>HATCY</acronym>
  <official_title>A Phase III, Multicenter, Randomized Controlled Study to Compare Safety and Efficacy of a Haploidentical HSCT and Adjunctive Treatment With ATIR101, a T-lymphocyte Enriched Leukocyte Preparation Depleted ex Vivo of Host Alloreactive T-cells, Versus a Haploidentical HSCT With Post-transplant Cyclophosphamide in Patients With a Hematologic Malignancy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Kiadis Pharma</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Kiadis Pharma</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The primary objective of this study is to compare safety and efficacy of a haploidentical&#xD;
      T-cell depleted HSCT and adjunctive treatment with ATIR101 versus a haploidentical T cell&#xD;
      replete HSCT with post-transplant administration of high dose cyclophosphamide (PTCy) in&#xD;
      patients with a hematologic malignancy. An additional objective of the study is to compare&#xD;
      the effect of the two treatments on quality of life.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Study CR-AIR-009 is a Phase III randomized controlled multicenter open-label study comparing&#xD;
      two parallel groups. After signing informed consent, a total of 250 patients will be&#xD;
      randomized in a 1:1 fashion to receive either a T-cell depleted hematopoietic stem cell&#xD;
      transplantation (HSCT; CD34 selection) from a related, haploidentical donor, followed by&#xD;
      ATIR101 infusion, or a T-cell replete HSCT, followed by a high dose of post-transplant&#xD;
      cyclophosphamide (PTCy).&#xD;
&#xD;
      Randomization will use minimization to balance treatment groups with respect to underlying&#xD;
      disease (AML, ALL, or MDS), Disease Risk Index (DRI; intermediate risk, high risk, or very&#xD;
      high risk) and center. A stochastic treatment allocation procedure will be used so that the&#xD;
      treatment assignment is random for all patients entered in the study.&#xD;
&#xD;
      Patients randomized in the ATIR101 group will receive a single ATIR101 dose of 2×10E6 viable&#xD;
      T-cells/kg between 28 and 32 days after the HSCT. Patients randomized in the PTCy group will&#xD;
      receive cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT. All patients will be&#xD;
      followed up for at least 24 months post HSCT.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Insufficient efficacy, terminated by Sponsor&#xD;
  </why_stopped>
  <start_date type="Actual">November 29, 2017</start_date>
  <completion_date type="Actual">December 17, 2021</completion_date>
  <primary_completion_date type="Actual">November 9, 2021</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Prevention</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Graft-versus-host Disease-free, Relapse-free Survival (GRFS)</measure>
    <time_frame>24 months post-HSCT</time_frame>
    <description>Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Overall Survival (OS)</measure>
    <time_frame>24 months post-HSCT</time_frame>
    <description>OS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Progression-free Survival (PFS)</measure>
    <time_frame>24 months post-HSCT</time_frame>
    <description>Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Relapse-related Mortality (RRM)</measure>
    <time_frame>Through study completion, at least two years post HSCT</time_frame>
    <description>Time from randomization to death due to disease relapse or disease progression</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Transplant-related Mortality (TRM)</measure>
    <time_frame>24 months post-HSCT</time_frame>
    <description>Defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Immune Reconstitution</measure>
    <time_frame>Through study completion, at least two years post HSCT</time_frame>
    <description>Time to CD3+ &gt; 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)</description>
  </other_outcome>
  <other_outcome>
    <measure>Cumulative Incidence of Grade II-IV and Grade III-IV Acute Graft-versus-host-disease (GVHD)</measure>
    <time_frame>Through study completion, at least two years post HSCT</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Cumulative Incidence of Moderate/Severe Chronic GVHD</measure>
    <time_frame>Through study completion, at least two years post HSCT</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Cumulative Incidence of Chronic GVHD Requiring Systemic Immunosuppressive Treatment</measure>
    <time_frame>Through study completion, at least two years post HSCT</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Duration of GVHD Episodes</measure>
    <time_frame>Through study completion, at least two years post HSCT</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Cumulative Incidence of NCI CTCAE Grade 2-5 and Grade 3-5 Infections</measure>
    <time_frame>Until 2 years after the HSCT</time_frame>
    <description>Viral, fungal, and bacterial infections</description>
  </other_outcome>
  <other_outcome>
    <measure>Cumulative Incidence of NCI CTCAE Grade 3-5 Adverse Events</measure>
    <time_frame>Until 2 years after the HSCT</time_frame>
    <description>Viral, fungal, and bacterial infections</description>
  </other_outcome>
  <other_outcome>
    <measure>FACT-BMT Total Score (Change From Screening)</measure>
    <time_frame>Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)</time_frame>
    <description>Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)</description>
  </other_outcome>
  <other_outcome>
    <measure>SF-36 Total Score (Change From Screening)</measure>
    <time_frame>Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)</time_frame>
    <description>Quality of life: Short Form 36-item health survey (SF-36)</description>
  </other_outcome>
  <other_outcome>
    <measure>MDASI Total Score (Change From Screening)</measure>
    <time_frame>Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)</time_frame>
    <description>Quality of life: MD Anderson Symptom Inventory (MDASI)</description>
  </other_outcome>
  <other_outcome>
    <measure>EQ-5D-5L (Change From Screening)</measure>
    <time_frame>Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)</time_frame>
    <description>Quality of life: EQ-5D-5L</description>
  </other_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">63</enrollment>
  <condition>Acute Myeloid Leukemia</condition>
  <condition>Acute Lymphoblastic Leukemia</condition>
  <condition>Myelodysplastic Syndrome</condition>
  <arm_group>
    <arm_group_label>ATIR101</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PTCy</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>ATIR101</intervention_name>
    <description>ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)</description>
    <arm_group_label>ATIR101</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Cyclophosphamide</intervention_name>
    <description>High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)</description>
    <arm_group_label>PTCy</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>T-cell depleted HSCT from a related, haploidentical donor</intervention_name>
    <description>T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
    <arm_group_label>ATIR101</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>T-cell replete HSCT from a related, haploidentical donor</intervention_name>
    <description>T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
    <arm_group_label>PTCy</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Any of the following hematologic malignancies:&#xD;
&#xD;
               -  Acute myeloid leukemia (AML) in first cytomorphological remission (with &lt; 5%&#xD;
                  blasts in the bone marrow) with Disease Risk Index (DRI) intermediate or above,&#xD;
                  or in second or higher cytomorphological remission (with &lt; 5% blasts in the bone&#xD;
                  marrow)&#xD;
&#xD;
               -  Acute lymphoblastic leukemia (ALL) in first or higher remission (with &lt; 5% blasts&#xD;
                  in the bone marrow)&#xD;
&#xD;
               -  Myelodysplastic syndrome (MDS): transfusion-dependent (requiring at least one&#xD;
                  transfusion per month), or intermediate or higher Revised International&#xD;
                  Prognostic Scoring System (IPSS-R) risk group&#xD;
&#xD;
          -  Clinical justification of allogeneic stem cell transplantation where a suitable HLA&#xD;
             matched sibling or unrelated donor is unavailable in a timely manner&#xD;
&#xD;
          -  Availability of a related haploidentical donor with one fully shared haplotype and 2&#xD;
             to 4 mismatches at the HLA-A, -B, -C, and -DRB1 loci of the unshared haplotype, as&#xD;
             determined by high resolution human leukocyte antigen (HLA)-typing&#xD;
&#xD;
          -  Karnofsky Performance Status (KPS) ≥ 70%&#xD;
&#xD;
          -  Male or female, age ≥ 18 years and ≤ 70 years. Patients aged ≥ 65 years must have a&#xD;
             Sorror score ≤ 3&#xD;
&#xD;
          -  Patient weight ≥ 25 kg and ≤ 130 kg&#xD;
&#xD;
          -  Availability of a donor aged ≥ 16 years and ≤ 75 years who is eligible according to&#xD;
             local requirements and regulations. Donors aged &lt; 16 years are allowed if they are the&#xD;
             only option for an HSCT, if they are permitted by local regulations, and if the&#xD;
             IRB/IEC approves participation in the study.&#xD;
&#xD;
          -  For females of childbearing potential who are sexually active and males who have&#xD;
             sexual contact with a female of childbearing potential: willingness to use of reliable&#xD;
             methods of contraception (oral contraceptives, intrauterine device, hormone implants,&#xD;
             contraceptive injection or abstinence) during study participation&#xD;
&#xD;
          -  Given written informed consent (patient and donor)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of chronic myelomonocytic leukemia (CMML)&#xD;
&#xD;
          -  Availability of a suitable HLA-matched sibling or unrelated donor in a donor search&#xD;
&#xD;
          -  Prior allogeneic hematopoietic stem cell transplantation&#xD;
&#xD;
          -  Diffusing capacity for carbon monoxide (hemoglobin corrected DLCO) &lt; 50% predicted&#xD;
&#xD;
          -  Left ventricular ejection fraction &lt; 45% (evaluated by echocardiogram or MUGA scan)&#xD;
&#xD;
          -  Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) &gt; 2.5 × upper&#xD;
             limit of normal (CTCAE grade 2)&#xD;
&#xD;
          -  Creatinine clearance &lt; 50 ml/min (calculated or measured)&#xD;
&#xD;
          -  Positive pregnancy test or breastfeeding of patient or donor (women of childbearing&#xD;
             age only)&#xD;
&#xD;
          -  Estimated probability of surviving less than 3 months&#xD;
&#xD;
          -  Known allergy to any of the components of ATIR101 (e.g., dimethyl sulfoxide)&#xD;
&#xD;
          -  Known hypersensitivity to cyclophosphamide or any of its metabolites&#xD;
&#xD;
          -  Any contraindication for GVHD prophylaxis with mycophenolate mofetil, cyclosporine A,&#xD;
             or tacrolimus&#xD;
&#xD;
          -  Known presence of HLA antibodies against the non-shared donor haplotype&#xD;
&#xD;
          -  Positive viral test of the patient or donor for human immunodeficiency virus (HIV)-1,&#xD;
             HIV-2, hepatitis B virus (HBV), hepatitis C virus (HCV), Treponema pallidum, human&#xD;
             T-lymphotropic virus (HTLV)-1 (if tested), HTLV-2 (if tested), West Nile virus (WNV;&#xD;
             if tested), or Zika virus (if tested)&#xD;
&#xD;
          -  Any other condition that, in the opinion of the investigator, makes the patient or&#xD;
             donor ineligible for the study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Denis Claude Roy, Prof MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Research Center and Cellular Therapy Laboratory, Maisonneuve-Rosemont Hospital (Montreal, Canada)</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Stephan Mielke, Prof MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Centre for Allogeneic Stem Cell Transplantation, Karolinska University Hospital (Stockholm, Sweden)</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>City of Hope National Medical Center</name>
      <address>
        <city>Duarte</city>
        <state>California</state>
        <zip>91010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Moores UC San Diego Cancer Center</name>
      <address>
        <city>La Jolla</city>
        <state>California</state>
        <zip>92037-0698</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UCLA Center for Health Sciences</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90095</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stanford University School of Medicine</name>
      <address>
        <city>Stanford</city>
        <state>California</state>
        <zip>94305</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Emory University</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Kansas Cancer Center</name>
      <address>
        <city>Westwood</city>
        <state>Kansas</state>
        <zip>66205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Massachusetts General Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Michigan</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109-1274</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Weill Cornell Medical College</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10021</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Columbia University Medical Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10032</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stony Brook University Hospital</name>
      <address>
        <city>Stony Brook</city>
        <state>New York</state>
        <zip>11794</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oregon Health &amp; Science University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitair Ziekenhuis Antwerpen</name>
      <address>
        <city>Antwerp</city>
        <zip>2650</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Algemeen Ziekenhuis Sint-Jan</name>
      <address>
        <city>Brugge</city>
        <zip>8000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Institut Jules Bordet</name>
      <address>
        <city>Brussels</city>
        <zip>1000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitair Ziekenhuis Gasthuisberg</name>
      <address>
        <city>Leuven</city>
        <zip>3000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre Hospitalier Universitaire de Liège</name>
      <address>
        <city>Liège</city>
        <zip>4000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Maisonneuve-Rosemont Hospital</name>
      <address>
        <city>Montreal</city>
        <state>Quebec</state>
        <zip>H1T 2M4</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital Centre Zagreb</name>
      <address>
        <city>Zagreb</city>
        <zip>10000</zip>
        <country>Croatia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>APHP Hospital Saint Louis</name>
      <address>
        <city>Paris</city>
        <zip>75475</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital Frankfurt, Goethe University</name>
      <address>
        <city>Frankfurt</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Medical Center Mainz</name>
      <address>
        <city>Mainz</city>
        <zip>55131</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ludwig-Maximilians-University Hospital of Munich-Grosshadern</name>
      <address>
        <city>Munich</city>
        <zip>81377</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Würzburg</name>
      <address>
        <city>Würzburg</city>
        <zip>97080</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rambam Medical Center</name>
      <address>
        <city>Haifa</city>
        <zip>3109601</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hadassah Medical Center &amp; Hadassah Hospital Ein Karem</name>
      <address>
        <city>Jerusalem</city>
        <zip>91120</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sourasky Medical Center &amp; Tel Aviv University</name>
      <address>
        <city>Tel Aviv</city>
        <zip>6423906</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Chaim Sheba Medical Center</name>
      <address>
        <city>Tel-Hashomer</city>
        <zip>5265601</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Milano Hospital, Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico</name>
      <address>
        <city>Milano</city>
        <zip>20122</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fondazione IRCCS Policlinico San Matteo</name>
      <address>
        <city>Pavia</city>
        <zip>27100</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Academisch Ziekenhuis Maastricht</name>
      <address>
        <city>Maastricht</city>
        <zip>6229 HX</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Faculdade de Medicina da Universidade de Lisboa</name>
      <address>
        <city>Lisboa</city>
        <zip>1649-028</zip>
        <country>Portugal</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital Barcelona Vall d' Hebron</name>
      <address>
        <city>Barcelona</city>
        <zip>08035</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Puerta de Hierro Majadahonda</name>
      <address>
        <city>Madrid</city>
        <zip>28220</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UGC Hematología y Hemoterapia</name>
      <address>
        <city>Sevilla</city>
        <zip>41013</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Servicio de Hematología Hospital, Universitari I politècnic La Fe</name>
      <address>
        <city>Valencia</city>
        <zip>46026</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Karolinska University Hospital</name>
      <address>
        <city>Stockholm</city>
        <zip>SE-141 86</zip>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Heartlands Hospital</name>
      <address>
        <city>Birmingham</city>
        <zip>B9 5SS</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St James University Hospital</name>
      <address>
        <city>Leeds</city>
        <zip>LS9 7TF</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Liverpool University Hospital</name>
      <address>
        <city>Liverpool</city>
        <zip>L7 8XP</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hammersmith Hospital</name>
      <address>
        <city>London</city>
        <zip>W12 0HS</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Manchester Royal Infirmary</name>
      <address>
        <city>Manchester</city>
        <zip>M13 9WL</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Belgium</country>
    <country>Canada</country>
    <country>Croatia</country>
    <country>France</country>
    <country>Germany</country>
    <country>Israel</country>
    <country>Italy</country>
    <country>Netherlands</country>
    <country>Portugal</country>
    <country>Spain</country>
    <country>Sweden</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>April 2022</verification_date>
  <study_first_submitted>December 19, 2016</study_first_submitted>
  <study_first_submitted_qc>December 19, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 21, 2016</study_first_posted>
  <results_first_submitted>March 29, 2022</results_first_submitted>
  <results_first_submitted_qc>April 28, 2022</results_first_submitted_qc>
  <results_first_posted type="Actual">May 24, 2022</results_first_posted>
  <last_update_submitted>April 28, 2022</last_update_submitted>
  <last_update_submitted_qc>April 28, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">May 24, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Haploidentical stem cell transplantation</keyword>
  <keyword>Graft-versus-host disease</keyword>
  <keyword>Immune reconstitution</keyword>
  <keyword>Alloreactive T-cells</keyword>
  <keyword>Photodynamic treatment</keyword>
  <keyword>Hematologic malignancy</keyword>
  <keyword>Transplant-related mortality</keyword>
  <keyword>Overall survival</keyword>
  <keyword>GRFS</keyword>
  <keyword>GVHD</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Precursor Cell Lymphoblastic Leukemia-Lymphoma</mesh_term>
    <mesh_term>Myelodysplastic Syndromes</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cyclophosphamide</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>August 23, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/54/NCT02999854/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>December 13, 2021</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/54/NCT02999854/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>ATIR101</title>
          <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
        </group>
        <group group_id="P2">
          <title>PTCy</title>
          <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Active Study Phase (up to 18 Months)</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="32"/>
                <participants group_id="P2" count="31"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="8"/>
                <participants group_id="P2" count="22"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="24"/>
                <participants group_id="P2" count="9"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Screen failures</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Discontinued before HSCT</title>
              <participants_list>
                <participants group_id="P1" count="8"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Death</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
                <participants group_id="P2" count="5"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Completed 2 year follow-up</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>End of study after HSCT but before treatment</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
        <period>
          <title>Long Term Safety FU (up to 24 Months)</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="8"/>
                <participants group_id="P2" count="22"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="5"/>
                <participants group_id="P2" count="19"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="3"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Death</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>ATIR101</title>
          <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
        </group>
        <group group_id="B2">
          <title>PTCy</title>
          <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
        </group>
        <group group_id="B3">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="16"/>
            <count group_id="B2" value="27"/>
            <count group_id="B3" value="43"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="36.2" spread="9.63"/>
                    <measurement group_id="B2" value="32.8" spread="13.20"/>
                    <measurement group_id="B3" value="34.5" spread="11.42"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="10"/>
                    <measurement group_id="B2" value="20"/>
                    <measurement group_id="B3" value="30"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                    <measurement group_id="B2" value="7"/>
                    <measurement group_id="B3" value="13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="12"/>
                    <measurement group_id="B2" value="22"/>
                    <measurement group_id="B3" value="34"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="14"/>
                    <measurement group_id="B2" value="23"/>
                    <measurement group_id="B3" value="37"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Hematologic malignancy</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Acute lymphatic leukemia (ALL)</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="6"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Acute myeloid leukemia (AML)</title>
                  <measurement_list>
                    <measurement group_id="B1" value="9"/>
                    <measurement group_id="B2" value="18"/>
                    <measurement group_id="B3" value="27"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Myelodysplastic syndrome (MDS)</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="7"/>
                    <measurement group_id="B3" value="10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Graft-versus-host Disease-free, Relapse-free Survival (GRFS)</title>
        <description>Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.</description>
        <time_frame>24 months post-HSCT</time_frame>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Graft-versus-host Disease-free, Relapse-free Survival (GRFS)</title>
          <description>Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.</description>
          <units>Percentage of participants</units>
          <param>Number</param>
          <dispersion>95% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="16"/>
                <count group_id="O2" value="27"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="25" lower_limit="8" upper_limit="47"/>
                    <measurement group_id="O2" value="62" lower_limit="41" upper_limit="78"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Overall Survival (OS)</title>
        <description>OS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.</description>
        <time_frame>24 months post-HSCT</time_frame>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Overall Survival (OS)</title>
          <description>OS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.</description>
          <units>percentage of participants</units>
          <param>Number</param>
          <dispersion>95% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="16"/>
                <count group_id="O2" value="27"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="49" lower_limit="24" upper_limit="71"/>
                    <measurement group_id="O2" value="77" lower_limit="55" upper_limit="89"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Progression-free Survival (PFS)</title>
        <description>Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.</description>
        <time_frame>24 months post-HSCT</time_frame>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Progression-free Survival (PFS)</title>
          <description>Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.</description>
          <units>percentage of participants</units>
          <param>Number</param>
          <dispersion>95% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="16"/>
                <count group_id="O2" value="27"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="44" lower_limit="20" upper_limit="66"/>
                    <measurement group_id="O2" value="73" lower_limit="52" upper_limit="86"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Relapse-related Mortality (RRM)</title>
        <description>Time from randomization to death due to disease relapse or disease progression</description>
        <time_frame>Through study completion, at least two years post HSCT</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Relapse-related Mortality (RRM)</title>
          <description>Time from randomization to death due to disease relapse or disease progression</description>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Transplant-related Mortality (TRM)</title>
        <description>Defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.</description>
        <time_frame>24 months post-HSCT</time_frame>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Transplant-related Mortality (TRM)</title>
          <description>Defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.</description>
          <units>percentage of participants</units>
          <param>Number</param>
          <dispersion>95% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="16"/>
                <count group_id="O2" value="27"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="44" lower_limit="19" upper_limit="66"/>
                    <measurement group_id="O2" value="15" lower_limit="5" upper_limit="31"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Immune Reconstitution</title>
        <description>Time to CD3+ &gt; 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)</description>
        <time_frame>Through study completion, at least two years post HSCT</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Immune Reconstitution</title>
          <description>Time to CD3+ &gt; 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)</description>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Cumulative Incidence of Grade II-IV and Grade III-IV Acute Graft-versus-host-disease (GVHD)</title>
        <time_frame>Through study completion, at least two years post HSCT</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Cumulative Incidence of Grade II-IV and Grade III-IV Acute Graft-versus-host-disease (GVHD)</title>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Cumulative Incidence of Moderate/Severe Chronic GVHD</title>
        <time_frame>Through study completion, at least two years post HSCT</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Cumulative Incidence of Moderate/Severe Chronic GVHD</title>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Cumulative Incidence of Chronic GVHD Requiring Systemic Immunosuppressive Treatment</title>
        <time_frame>Through study completion, at least two years post HSCT</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Cumulative Incidence of Chronic GVHD Requiring Systemic Immunosuppressive Treatment</title>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Duration of GVHD Episodes</title>
        <time_frame>Through study completion, at least two years post HSCT</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Duration of GVHD Episodes</title>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Cumulative Incidence of NCI CTCAE Grade 2-5 and Grade 3-5 Infections</title>
        <description>Viral, fungal, and bacterial infections</description>
        <time_frame>Until 2 years after the HSCT</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Cumulative Incidence of NCI CTCAE Grade 2-5 and Grade 3-5 Infections</title>
          <description>Viral, fungal, and bacterial infections</description>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Cumulative Incidence of NCI CTCAE Grade 3-5 Adverse Events</title>
        <description>Viral, fungal, and bacterial infections</description>
        <time_frame>Until 2 years after the HSCT</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>Cumulative Incidence of NCI CTCAE Grade 3-5 Adverse Events</title>
          <description>Viral, fungal, and bacterial infections</description>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>FACT-BMT Total Score (Change From Screening)</title>
        <description>Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)</description>
        <time_frame>Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>FACT-BMT Total Score (Change From Screening)</title>
          <description>Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)</description>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>SF-36 Total Score (Change From Screening)</title>
        <description>Quality of life: Short Form 36-item health survey (SF-36)</description>
        <time_frame>Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>SF-36 Total Score (Change From Screening)</title>
          <description>Quality of life: Short Form 36-item health survey (SF-36)</description>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>MDASI Total Score (Change From Screening)</title>
        <description>Quality of life: MD Anderson Symptom Inventory (MDASI)</description>
        <time_frame>Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>MDASI Total Score (Change From Screening)</title>
          <description>Quality of life: MD Anderson Symptom Inventory (MDASI)</description>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>EQ-5D-5L (Change From Screening)</title>
        <description>Quality of life: EQ-5D-5L</description>
        <time_frame>Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)</time_frame>
        <population>Study was pre-maturely terminated. Data not collected.</population>
        <group_list>
          <group group_id="O1">
            <title>ATIR101</title>
            <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
          <group group_id="O2">
            <title>PTCy</title>
            <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
          </group>
        </group_list>
        <measure>
          <title>EQ-5D-5L (Change From Screening)</title>
          <description>Quality of life: EQ-5D-5L</description>
          <population>Study was pre-maturely terminated. Data not collected.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Long term safety follow-up, 24 months.</time_frame>
      <desc>Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded.&#xD;
(63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat [ITT] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).</desc>
      <group_list>
        <group group_id="E1">
          <title>ATIR101</title>
          <description>T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT&#xD;
ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)&#xD;
T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
        </group>
        <group group_id="E2">
          <title>PTCy</title>
          <description>T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT&#xD;
Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)&#xD;
T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="6" subjects_at_risk="27"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="13" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="19" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Blood and lymphatic system disorders</sub_title>
                <counts group_id="E1" subjects_affected="5" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="5" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Cardiac disorders</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="5" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Gastrointestinal disorders</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>General disorders and administration site conditions</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="6" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Infections and infestations</sub_title>
                <counts group_id="E1" subjects_affected="10" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="12" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Injury, poisoning and procedural complications</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Investigations</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Metabolism and nutrition disorders</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Nervous system disorders</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Respiratory, thoracic and mediastinal disorders</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="16"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="27"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Klaudia Traudtner, MD. Head of Safety and Pharmacovigilance.</name_or_title>
      <organization>Kiadis Pharma Netherlands B.V</organization>
      <phone>+31 (0)20 240 52 50</phone>
      <email>k.traudtner@kiadis.com</email>
    </point_of_contact>
  </clinical_results>
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT02999854
org study id: CR-AIR-009
secondary id: 2016-004672-21
nct id: NCT02999854
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The primary objective of this study is to compare safety and efficacy of a haploidentical
      T-cell depleted HSCT and adjunctive treatment with ATIR101 versus a haploidentical T cell
      replete HSCT with post-transplant administration of high dose cyclophosphamide (PTCy) in
      patients with a hematologic malignancy. An additional objective of the study is to compare
      the effect of the two treatments on quality of life.
Study CR-AIR-009 is a Phase III randomized controlled multicenter open-label study comparing
      two parallel groups. After signing informed consent, a total of 250 patients will be
      randomized in a 1:1 fashion to receive either a T-cell depleted hematopoietic stem cell
      transplantation (HSCT; CD34 selection) from a related, haploidentical donor, followed by
      ATIR101 infusion, or a T-cell replete HSCT, followed by a high dose of post-transplant
      cyclophosphamide (PTCy).

      Randomization will use minimization to balance treatment groups with respect to underlying
      disease (AML, ALL, or MDS), Disease Risk Index (DRI; intermediate risk, high risk, or very
      high risk) and center. A stochastic treatment allocation procedure will be used so that the
      treatment assignment is random for all patients entered in the study.

      Patients randomized in the ATIR101 group will receive a single ATIR101 dose of 2×10E6 viable
      T-cells/kg between 28 and 32 days after the HSCT. Patients randomized in the PTCy group will
      receive cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT. All patients will be
      followed up for at least 24 months post HSCT.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Prevention
masking: None (Open Label)
measure: Graft-versus-host Disease-free, Relapse-free Survival (GRFS)
time frame: 24 months post-HSCT
description: Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.
measure: Overall Survival (OS)
time frame: 24 months post-HSCT
description: OS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.
measure: Progression-free Survival (PFS)
time frame: 24 months post-HSCT
description: Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
measure: Relapse-related Mortality (RRM)
time frame: Through study completion, at least two years post HSCT
description: Time from randomization to death due to disease relapse or disease progression
measure: Transplant-related Mortality (TRM)
time frame: 24 months post-HSCT
description: Defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
measure: Immune Reconstitution
time frame: Through study completion, at least two years post HSCT
description: Time to CD3+ > 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)
measure: Cumulative Incidence of Grade II-IV and Grade III-IV Acute Graft-versus-host-disease (GVHD)
time frame: Through study completion, at least two years post HSCT
measure: Cumulative Incidence of Moderate/Severe Chronic GVHD
time frame: Through study completion, at least two years post HSCT
measure: Cumulative Incidence of Chronic GVHD Requiring Systemic Immunosuppressive Treatment
time frame: Through study completion, at least two years post HSCT
measure: Duration of GVHD Episodes
time frame: Through study completion, at least two years post HSCT
measure: Cumulative Incidence of NCI CTCAE Grade 2-5 and Grade 3-5 Infections
time frame: Until 2 years after the HSCT
description: Viral, fungal, and bacterial infections
measure: Cumulative Incidence of NCI CTCAE Grade 3-5 Adverse Events
time frame: Until 2 years after the HSCT
description: Viral, fungal, and bacterial infections
measure: FACT-BMT Total Score (Change From Screening)
time frame: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)
description: Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)
measure: SF-36 Total Score (Change From Screening)
time frame: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)
description: Quality of life: Short Form 36-item health survey (SF-36)
measure: MDASI Total Score (Change From Screening)
time frame: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)
description: Quality of life: MD Anderson Symptom Inventory (MDASI)
measure: EQ-5D-5L (Change From Screening)
time frame: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)
description: Quality of life: EQ-5D-5L
arm group label: ATIR101
arm group type: Experimental
description: T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
arm group label: PTCy
arm group type: Active Comparator
description: T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
intervention type: Biological
intervention name: ATIR101
description: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
arm group label: ATIR101
intervention type: Drug
intervention name: Cyclophosphamide
description: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
arm group label: PTCy
intervention type: Procedure
intervention name: T-cell depleted HSCT from a related, haploidentical donor
description: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
arm group label: ATIR101
intervention type: Procedure
intervention name: T-cell replete HSCT from a related, haploidentical donor
description: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
arm group label: PTCy
criteria: 
gender: All
minimum age: 18 Years
maximum age: 70 Years
healthy volunteers: No
last name: Denis Claude Roy, Prof MD
role: Principal Investigator
affiliation: Research Center and Cellular Therapy Laboratory, Maisonneuve-Rosemont Hospital (Montreal, Canada)
last name: Stephan Mielke, Prof MD
role: Principal Investigator
affiliation: Centre for Allogeneic Stem Cell Transplantation, Karolinska University Hospital (Stockholm, Sweden)
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country: Belgium
country: Canada
country: Croatia
country: France
country: Germany
country: Israel
country: Italy
country: Netherlands
country: Portugal
country: Spain
country: Sweden
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Leukemia
mesh term: Precursor Cell Lymphoblastic Leukemia-Lymphoma
mesh term: Myelodysplastic Syndromes
mesh term: Cyclophosphamide
sharing ipd: No
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certain agreements: 
point of contact: 