<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/NCT04473911</url>
  </required_header>
  <id_info>
    <org_study_id>19-336</org_study_id>
    <nct_id>NCT04473911</nct_id>
  </id_info>
  <brief_title>Haplo Peripheral Blood Sct In GVHD Prevention</brief_title>
  <official_title>Reduced Intensity Haploidentical Peripheral Blood Stem Cell Transplantation With Post-transplant Cyclophosphamide and Sirolimus/Mycophenolate Mofetil/RGI-2001 Based GVHD Prevention: a Pilot Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Zachariah Michael DeFilipp</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Regimmune Corporation</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Massachusetts General Hospital</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>
      This research study is studying the RGI-2001 for preventing Graft-vs-Host Disease (GVHD) in&#xD;
      people with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic&#xD;
      syndrome (MDS), myeloproliferative disorders (MPN), chronic myelomonocytic leukemic (CMML),&#xD;
      chemosensitive hodgkin lymphoma (HL), or Non-Hodgkin lymphoma (NHL).who will have a blood&#xD;
      stem cell transplantation.&#xD;
&#xD;
        -  GVHD is a condition in which cells from the donor's tissue attack the organs.&#xD;
&#xD;
        -  RGI-2001 is an investigational treatment&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      -  This is a pilot study in subjects undergoing reduced-intensity haploidentical peripheral&#xD;
           blood stem cell transplantation who will receive graft-versus-host disease prevention&#xD;
           with post-transplant cyclophosphamide, followed by sirolimus, mycophenolate mofetil, and&#xD;
           RGI-2001.&#xD;
&#xD;
        -  The research study procedures include screening for eligibility and study treatment&#xD;
           including evaluations and follow up visits.&#xD;
&#xD;
        -  The standard of care drugs of fludarabine, cyclophosphamide, melphalan, radiation,&#xD;
           sirolimus, and mycophenolate mofetil are all FDA approved.&#xD;
&#xD;
             -  Eligible Participants will be placed in 1 of 2 groups, per physicians discretion:&#xD;
&#xD;
             -  Regimen #1 :&#xD;
&#xD;
                  -  Before stem cell transplant:Fludarabine + Cyclophosphamide + Radiation&#xD;
&#xD;
                  -  After stem cell transplant: Cyclophosphamide + Sirolimus +Mycophenolate&#xD;
                     mofetil + RGI-2001&#xD;
&#xD;
             -  Regimen #2&#xD;
&#xD;
                  -  Before stem cell transplant: fludarabine + melphalan + radiation&#xD;
&#xD;
                  -  After stem cell transplant: cyclophosphamide + sirolimus +Mycophenolate&#xD;
                     mofetil + RGI-2001&#xD;
&#xD;
        -  A total of 20 participants will be enrolled to this trial&#xD;
&#xD;
        -  The U.S. Food and Drug Administration (FDA) has not approved RGI-2001 as a treatment for&#xD;
           any disease.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">August 14, 2020</start_date>
  <completion_date type="Anticipated">December 2023</completion_date>
  <primary_completion_date type="Anticipated">December 2022</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of patients achieving successful donor engraftment</measure>
    <time_frame>60 Days</time_frame>
    <description>(absolute neutrophil count &gt; 500/uL and ≥ 90% donor cell chimerism)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>100-day non-relapse mortality (NRM) rate.</measure>
    <time_frame>100 Days</time_frame>
    <description>The regimen will be considered as safe if 100d NRM rate is &lt;=5%, and not safe if 100d NRM rate is ≥25%.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">20</enrollment>
  <condition>GVHD</condition>
  <condition>AML</condition>
  <condition>ALL</condition>
  <condition>MDS</condition>
  <condition>MPN</condition>
  <condition>CMML</condition>
  <condition>Hodgkin Lymphoma</condition>
  <condition>Non Hodgkin Lymphoma</condition>
  <condition>Blood Stem Cell Transplant Failure</condition>
  <condition>Graft Vs Host Disease</condition>
  <condition>Acute Myeloid Leukemia</condition>
  <condition>Acute Lymphoblastic Leukemia</condition>
  <condition>Myelodysplastic Syndromes</condition>
  <condition>Myeloproliferative Disorder</condition>
  <condition>Chronic Myelomonocytic Leukemia</condition>
  <condition>Chemosensitive Hodgkin Lymphoma</condition>
  <arm_group>
    <arm_group_label>Regimen 1: Fludarabine, Cyclophosphamide, and TBI</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>-. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician&#xD;
Pre- stem cell transplant:&#xD;
Fludarabine predetermined dose, intravenously, 4 times per cycle&#xD;
Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion&#xD;
Total body irradiation (TBI) once during treatment cycle&#xD;
Post stem cell transplant:&#xD;
Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion&#xD;
Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral.&#xD;
Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle&#xD;
RGI-2001: IV, predetermined dose, weekly to 6 total doses</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Regimen 2: Fludarabine, Melphalan, and TBI</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>-. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician&#xD;
Pre- stem cell transplant:&#xD;
Fludarabine predetermined dose, intravenously 3 times per cycle&#xD;
Melphalan, infusion, determined dosage, once per cycle&#xD;
Total body irradiation (TBI) once per cycle.&#xD;
Post stem cell transplant&#xD;
Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion&#xD;
Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral:&#xD;
Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle&#xD;
RGI-2001: IV, predetermined dose, weekly to 6 total doses</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>FLUDARABINE</intervention_name>
    <description>predetermined dose, intravenously, a predetermined times per cycle Given in both pre stem cell and post stem cell cycles</description>
    <arm_group_label>Regimen 1: Fludarabine, Cyclophosphamide, and TBI</arm_group_label>
    <arm_group_label>Regimen 2: Fludarabine, Melphalan, and TBI</arm_group_label>
    <other_name>Fludara®</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>CYCLOPHOSPHAMIDE</intervention_name>
    <description>◦Cyclophosphamide predetermined dose, predetermined number of times in Given in pre-stem cell Regimen #1 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion</description>
    <arm_group_label>Regimen 1: Fludarabine, Cyclophosphamide, and TBI</arm_group_label>
    <other_name>Cytoxan®</other_name>
    <other_name>Neosar®</other_name>
  </intervention>
  <intervention>
    <intervention_type>Radiation</intervention_type>
    <intervention_name>TBI</intervention_name>
    <description>Total body irradiation (TBI) once per cycle.</description>
    <arm_group_label>Regimen 1: Fludarabine, Cyclophosphamide, and TBI</arm_group_label>
    <arm_group_label>Regimen 2: Fludarabine, Melphalan, and TBI</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Melphalan</intervention_name>
    <description>Melphalan, infusion, determined dosage, once per cycle</description>
    <arm_group_label>Regimen 2: Fludarabine, Melphalan, and TBI</arm_group_label>
    <other_name>Alkeran®</other_name>
    <other_name>L-PAM</other_name>
    <other_name>L-Sarcolysin</other_name>
    <other_name>Phenylalanine Mustard</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Sirolimus</intervention_name>
    <description>Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral: Please note that doses of sirolimus can be adjusted at the treating physician's discretion given the multiple drugs and other situations which affect its metabolism</description>
    <arm_group_label>Regimen 1: Fludarabine, Cyclophosphamide, and TBI</arm_group_label>
    <arm_group_label>Regimen 2: Fludarabine, Melphalan, and TBI</arm_group_label>
    <other_name>Rapamune</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Mycophenolate mofetil</intervention_name>
    <description>◦Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle</description>
    <arm_group_label>Regimen 1: Fludarabine, Cyclophosphamide, and TBI</arm_group_label>
    <arm_group_label>Regimen 2: Fludarabine, Melphalan, and TBI</arm_group_label>
    <other_name>CellCept</other_name>
    <other_name>Myfortic</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>RGI-2001</intervention_name>
    <description>IV, predetermined dose, weekly to 6 total doses</description>
    <arm_group_label>Regimen 1: Fludarabine, Cyclophosphamide, and TBI</arm_group_label>
    <arm_group_label>Regimen 2: Fludarabine, Melphalan, and TBI</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>CYCLOPHOSPHAMIDE</intervention_name>
    <description>◦Given in Post Stem Cell cycle of Regimen #1 and #2 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion</description>
    <arm_group_label>Regimen 1: Fludarabine, Cyclophosphamide, and TBI</arm_group_label>
    <arm_group_label>Regimen 2: Fludarabine, Melphalan, and TBI</arm_group_label>
    <other_name>Cytoxan®</other_name>
    <other_name>Neosar®</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Men or women ≥ 18 and ≤ 80 years old&#xD;
&#xD;
          -  Diagnosis of hematological malignancy:&#xD;
&#xD;
               -  Acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) in morphologic&#xD;
                  complete remission&#xD;
&#xD;
               -  Myelodysplastic syndrome (MDS), myeloproliferative disorders (MPN), or chronic&#xD;
                  myelomonocytic leukemic (CMML) with &lt; 5% blasts in blood or bone marrow&#xD;
&#xD;
               -  Chemosensitive Hodgkin lymphoma (HL) or Non-Hodgkin lymphoma (NHL)&#xD;
&#xD;
          -  Patients must be undergoing haploidentical allogeneic hematopoietic cell&#xD;
             transplantation, defined as 1st or 2nd degree relative with at least 5/10 matching at&#xD;
             HLA-A, -B, -C, DR, and DQ.&#xD;
&#xD;
          -  ECOG performance status ≤2&#xD;
&#xD;
          -  Patients with adequate physical function as measured by:&#xD;
&#xD;
               -  Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening&#xD;
                  fraction &gt;25%&#xD;
&#xD;
               -  Hepatic:&#xD;
&#xD;
                    -  Bilirubin ≤ 2.5 mg/dL, except for patients with Gilbert's syndrome or&#xD;
                       hemolysis&#xD;
&#xD;
                    -  ALT, AST, and Alkaline Phosphatase &lt; 5 x ULN&#xD;
&#xD;
               -  Renal: Serum creatinine within normal range, or if serum creatinine is outside&#xD;
                  normal range, then renal function (measured or estimated creatinine clearance or&#xD;
                  GFR) ≥ 40mL/min/1.73m2&#xD;
&#xD;
               -  Pulmonary: DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted&#xD;
&#xD;
          -  Ability to understand and the willingness to sign a written informed consent document&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Prior allogeneic hematopoietic stem cell transplantation. (Patients may have received&#xD;
             a prior autologous hematopoietic stem cell transplant.)&#xD;
&#xD;
          -  Participants who are receiving any other investigational agents within 14 days prior&#xD;
             to RGI-2001 dosing. Thus, participants must stop investigational agents by Day -9&#xD;
             prior to transplant.&#xD;
&#xD;
          -  Uncontrolled intercurrent illness including, but not limited to, symptomatic&#xD;
             congestive heart failure, recent myocardial infarction or stroke, unstable angina&#xD;
             pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would&#xD;
             limit compliance with study requirements.&#xD;
&#xD;
          -  Patients with active or uncontrolled bacterial, viral, or fungal infection(s)&#xD;
             requiring systemic therapy.&#xD;
&#xD;
          -  Planned use of prophylactic donor lymphocyte infusion (DLI) therapy.&#xD;
&#xD;
          -  Pregnant and breast-feeding women are ineligible because they are not eligible for&#xD;
             hematopoietic stem cell transplantation.&#xD;
&#xD;
          -  HIV-positive participants and patients with active Hepatitis B or C are ineligible&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Zachariah DeFilipp, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Massachusetts General Hospital</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Zachariah DeFilipp, MD</last_name>
    <phone>(617) 726-5765</phone>
    <email>zdefilipp@mgh.harvard.edu</email>
  </overall_contact>
  <location>
    <facility>
      <name>Massachusetts General Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Zachariah DeFilipp, MD</last_name>
      <phone>617-726-5765</phone>
    </contact>
    <investigator>
      <last_name>Zachariah DeFilipp, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2021</verification_date>
  <study_first_submitted>July 13, 2020</study_first_submitted>
  <study_first_submitted_qc>July 13, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 16, 2020</study_first_posted>
  <last_update_submitted>September 30, 2021</last_update_submitted>
  <last_update_submitted_qc>September 30, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">October 4, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Massachusetts General Hospital</investigator_affiliation>
    <investigator_full_name>Zachariah Michael DeFilipp</investigator_full_name>
    <investigator_title>Sponsor Investigator</investigator_title>
  </responsible_party>
  <keyword>GVHD</keyword>
  <keyword>AML</keyword>
  <keyword>ALL</keyword>
  <keyword>MDS</keyword>
  <keyword>MPN</keyword>
  <keyword>CMML</keyword>
  <keyword>Hodgkin Lymphoma</keyword>
  <keyword>Non Hodgkin Lymphoma</keyword>
  <keyword>Blood Stem Cell Transplant Failure</keyword>
  <keyword>Graft Vs Host Disease</keyword>
  <keyword>Acute Myeloid Leukemia</keyword>
  <keyword>Acute Lymphoblastic Leukemia</keyword>
  <keyword>Myelodysplastic Syndromes</keyword>
  <keyword>Myeloproliferative Disorder</keyword>
  <keyword>Chronic Myelomonocytic Leukemia</keyword>
  <keyword>Chemosensitive Hodgkin Lymphoma</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Lymphoma</mesh_term>
    <mesh_term>Leukemia</mesh_term>
    <mesh_term>Leukemia, Myeloid</mesh_term>
    <mesh_term>Leukemia, Myeloid, Acute</mesh_term>
    <mesh_term>Lymphoma, Non-Hodgkin</mesh_term>
    <mesh_term>Preleukemia</mesh_term>
    <mesh_term>Precursor Cell Lymphoblastic Leukemia-Lymphoma</mesh_term>
    <mesh_term>Leukemia, Lymphoid</mesh_term>
    <mesh_term>Hodgkin Disease</mesh_term>
    <mesh_term>Leukemia, Myelomonocytic, Chronic</mesh_term>
    <mesh_term>Leukemia, Myelomonocytic, Juvenile</mesh_term>
    <mesh_term>Myelodysplastic Syndromes</mesh_term>
    <mesh_term>Myeloproliferative Disorders</mesh_term>
    <mesh_term>Graft vs Host Disease</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Mycophenolic Acid</mesh_term>
    <mesh_term>Sirolimus</mesh_term>
    <mesh_term>Cyclophosphamide</mesh_term>
    <mesh_term>Melphalan</mesh_term>
    <mesh_term>Fludarabine</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to, please contact the Sponsor Investigator or designee]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.</ipd_description>
    <ipd_info_type>Study Protocol</ipd_info_type>
    <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
    <ipd_info_type>Informed Consent Form (ICF)</ipd_info_type>
    <ipd_time_frame>Data can be shared no earlier than 1 year following the date of publication</ipd_time_frame>
    <ipd_access_criteria>Contact the Partners Innovations team at http://www.partners.org/innovation</ipd_access_criteria>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT04473911
org study id: 19-336
nct id: NCT04473911
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This research study is studying the RGI-2001 for preventing Graft-vs-Host Disease (GVHD) in
      people with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic
      syndrome (MDS), myeloproliferative disorders (MPN), chronic myelomonocytic leukemic (CMML),
      chemosensitive hodgkin lymphoma (HL), or Non-Hodgkin lymphoma (NHL).who will have a blood
      stem cell transplantation.

        -  GVHD is a condition in which cells from the donor's tissue attack the organs.

        -  RGI-2001 is an investigational treatment
-  This is a pilot study in subjects undergoing reduced-intensity haploidentical peripheral
           blood stem cell transplantation who will receive graft-versus-host disease prevention
           with post-transplant cyclophosphamide, followed by sirolimus, mycophenolate mofetil, and
           RGI-2001.

        -  The research study procedures include screening for eligibility and study treatment
           including evaluations and follow up visits.

        -  The standard of care drugs of fludarabine, cyclophosphamide, melphalan, radiation,
           sirolimus, and mycophenolate mofetil are all FDA approved.

             -  Eligible Participants will be placed in 1 of 2 groups, per physicians discretion:

             -  Regimen #1 :

                  -  Before stem cell transplant:Fludarabine + Cyclophosphamide + Radiation

                  -  After stem cell transplant: Cyclophosphamide + Sirolimus +Mycophenolate
                     mofetil + RGI-2001

             -  Regimen #2

                  -  Before stem cell transplant: fludarabine + melphalan + radiation

                  -  After stem cell transplant: cyclophosphamide + sirolimus +Mycophenolate
                     mofetil + RGI-2001

        -  A total of 20 participants will be enrolled to this trial

        -  The U.S. Food and Drug Administration (FDA) has not approved RGI-2001 as a treatment for
           any disease.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of patients achieving successful donor engraftment
time frame: 60 Days
description: (absolute neutrophil count > 500/uL and ≥ 90% donor cell chimerism)
measure: 100-day non-relapse mortality (NRM) rate.
time frame: 100 Days
description: The regimen will be considered as safe if 100d NRM rate is <=5%, and not safe if 100d NRM rate is ≥25%.
arm group label: Regimen 1: Fludarabine, Cyclophosphamide, and TBI
arm group type: Experimental
description: -. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician
Pre- stem cell transplant:
Fludarabine predetermined dose, intravenously, 4 times per cycle
Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion
Total body irradiation (TBI) once during treatment cycle
Post stem cell transplant:
Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion
Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral.
Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle
RGI-2001: IV, predetermined dose, weekly to 6 total doses
arm group label: Regimen 2: Fludarabine, Melphalan, and TBI
arm group type: Experimental
description: -. Patients who meet eligibility criteria for the study will subsequently be enrolled for treatment. Two reduced intensity regimens will be allowed, according to the choice of the treating physician
Pre- stem cell transplant:
Fludarabine predetermined dose, intravenously 3 times per cycle
Melphalan, infusion, determined dosage, once per cycle
Total body irradiation (TBI) once per cycle.
Post stem cell transplant
Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion
Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral:
Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle
RGI-2001: IV, predetermined dose, weekly to 6 total doses
intervention type: Drug
intervention name: FLUDARABINE
description: predetermined dose, intravenously, a predetermined times per cycle Given in both pre stem cell and post stem cell cycles
arm group label: Regimen 1: Fludarabine, Cyclophosphamide, and TBI
arm group label: Regimen 2: Fludarabine, Melphalan, and TBI
other name: Fludara®
intervention type: Drug
intervention name: CYCLOPHOSPHAMIDE
description: ◦Cyclophosphamide predetermined dose, predetermined number of times in Given in pre-stem cell Regimen #1 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion
arm group label: Regimen 1: Fludarabine, Cyclophosphamide, and TBI
other name: Cytoxan®
other name: Neosar®
intervention type: Radiation
intervention name: TBI
description: Total body irradiation (TBI) once per cycle.
arm group label: Regimen 1: Fludarabine, Cyclophosphamide, and TBI
arm group label: Regimen 2: Fludarabine, Melphalan, and TBI
intervention type: Drug
intervention name: Melphalan
description: Melphalan, infusion, determined dosage, once per cycle
arm group label: Regimen 2: Fludarabine, Melphalan, and TBI
other name: Alkeran®
other name: L-PAM
other name: L-Sarcolysin
other name: Phenylalanine Mustard
intervention type: Drug
intervention name: Sirolimus
description: Sirolimus: Predetermined dosage, predetermined number of time in cycle, oral: Please note that doses of sirolimus can be adjusted at the treating physician's discretion given the multiple drugs and other situations which affect its metabolism
arm group label: Regimen 1: Fludarabine, Cyclophosphamide, and TBI
arm group label: Regimen 2: Fludarabine, Melphalan, and TBI
other name: Rapamune
intervention type: Drug
intervention name: Mycophenolate mofetil
description: ◦Mycophenolate mofetil, oral or iv(predetermined dose or IV TID (based upon actual body weight), at predetermined times per cycle
arm group label: Regimen 1: Fludarabine, Cyclophosphamide, and TBI
arm group label: Regimen 2: Fludarabine, Melphalan, and TBI
other name: CellCept
other name: Myfortic
intervention type: Drug
intervention name: RGI-2001
description: IV, predetermined dose, weekly to 6 total doses
arm group label: Regimen 1: Fludarabine, Cyclophosphamide, and TBI
arm group label: Regimen 2: Fludarabine, Melphalan, and TBI
intervention type: Drug
intervention name: CYCLOPHOSPHAMIDE
description: ◦Given in Post Stem Cell cycle of Regimen #1 and #2 Cyclophosphamide predetermined dose, predetermined number of times in cycle, intravenous infusion
arm group label: Regimen 1: Fludarabine, Cyclophosphamide, and TBI
arm group label: Regimen 2: Fludarabine, Melphalan, and TBI
other name: Cytoxan®
other name: Neosar®
criteria: 
gender: All
minimum age: 18 Years
maximum age: 80 Years
healthy volunteers: No
last name: Zachariah DeFilipp, MD
role: Principal Investigator
affiliation: Massachusetts General Hospital
last name: Zachariah DeFilipp, MD
phone: (617) 726-5765
email: zdefilipp@mgh.harvard.edu
facility: 
status: Recruiting
contact: 
investigator: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Massachusetts General Hospital
investigator full name: Zachariah Michael DeFilipp
investigator title: Sponsor Investigator
mesh term: Lymphoma
mesh term: Leukemia
mesh term: Leukemia, Myeloid
mesh term: Leukemia, Myeloid, Acute
mesh term: Lymphoma, Non-Hodgkin
mesh term: Preleukemia
mesh term: Precursor Cell Lymphoblastic Leukemia-Lymphoma
mesh term: Leukemia, Lymphoid
mesh term: Hodgkin Disease
mesh term: Leukemia, Myelomonocytic, Chronic
mesh term: Leukemia, Myelomonocytic, Juvenile
mesh term: Myelodysplastic Syndromes
mesh term: Myeloproliferative Disorders
mesh term: Graft vs Host Disease
mesh term: Mycophenolic Acid
mesh term: Sirolimus
mesh term: Cyclophosphamide
mesh term: Melphalan
mesh term: Fludarabine
sharing ipd: Yes
ipd description: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to, please contact the Sponsor Investigator or designee]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
ipd info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd info type: Informed Consent Form (ICF)
ipd time frame: Data can be shared no earlier than 1 year following the date of publication
ipd access criteria: Contact the Partners Innovations team at http://www.partners.org/innovation