<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/NCT04437771</url>
  </required_header>
  <id_info>
    <org_study_id>RP-L102-0116-LTFU</org_study_id>
    <nct_id>NCT04437771</nct_id>
  </id_info>
  <brief_title>Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy</brief_title>
  <official_title>Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Patients With Fanconi Anaemia Subtype A: FANCOLEN-I</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Rocket Pharmaceuticals Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Rocket Pharmaceuticals Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia&#xD;
      Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial. After&#xD;
      completion of the FANCOLEN-I study, eligible subjects will be followed for a total of 15&#xD;
      years post gene therapy treatment. No investigational drug product will be administered&#xD;
      during this study.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This long-term follow-up protocol will evaluate the long term safety and efficacy of the&#xD;
      infusion of autologous CD34+ cells transduced with lentiviral vector (LV) carrying the FANCA&#xD;
      gene.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Enrolling by invitation</overall_status>
  <start_date type="Actual">June 1, 2020</start_date>
  <completion_date type="Anticipated">January 30, 2035</completion_date>
  <primary_completion_date type="Anticipated">October 30, 2034</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Monitor long term safety of patients through blood laboratory evaluations and general health status</measure>
    <time_frame>15 years post-drug product infusion</time_frame>
    <description>Evaluate long term safety following infusion of hematopoietic cells transduced with therapeutic lentiviral vector (LV)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Long term genetic correction assessed in bone marrow and blood</measure>
    <time_frame>15 years post-drug product infusion</time_frame>
    <description>Determine long term persistence of therapeutic LV in hematopoietic cells in bone marrow and blood</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Replication competent lentivirus (RCL)</measure>
    <time_frame>15 years post-drug product infusion</time_frame>
    <description>Evaluate RCL in peripheral blood</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Insertion site analysis in blood</measure>
    <time_frame>15 years post-drug product infusion</time_frame>
    <description>Determine long term clonality</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Phenotypic correction</measure>
    <time_frame>15 years post-drug product infusion</time_frame>
    <description>Determine phenotypic correction of bone marrow and peripheral blood cells by resistance to DNA-damaging agents</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Assessment for Malignancies</measure>
    <time_frame>15 years post-drug product infusion</time_frame>
    <description>Monitor for incidence of hematologic malignancies and solid organ tumors</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Hematologic stabilization</measure>
    <time_frame>15 years post-drug product infusion</time_frame>
    <description>Monitor for long term stability and normalization of blood counts</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">9</enrollment>
  <condition>Fanconi Anemia Complementation Group A</condition>
  <condition>Fanconi Anemia</condition>
  <arm_group>
    <arm_group_label>Subjects with Fanconi Anaemia Subtype A (FA-A)</arm_group_label>
    <description>Subjects treated with ex vivo lentiviral gene therapy product in FANCOLEN-I trial and agree to participate in this long-term follow-up (LTFU) study</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Safety and efficacy assessments</intervention_name>
    <description>Long term disease and gene therapy specific safety evaluations and efficacy assessments</description>
    <arm_group_label>Subjects with Fanconi Anaemia Subtype A (FA-A)</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Subjects with Fanconi anemia subtype A who have been treated with ex vivo gene therapy&#xD;
        product in FANCOLEN-I study&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Enrolled in the FANCOLEN-I study&#xD;
&#xD;
          2. Treated with gene therapy in the FANCOLEN-I study&#xD;
&#xD;
          3. Able to adhere to the study visit schedule and protocol requirements&#xD;
&#xD;
          4. Provided written informed consent and, as applicable, assent to participate&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  There are no exclusion criteria for this study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Hospital Infantil Universitario Niño Jesús (HIUNJ)</name>
      <address>
        <city>Madrid</city>
        <zip>28009</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Spain</country>
  </location_countries>
  <verification_date>June 2020</verification_date>
  <study_first_submitted>June 15, 2020</study_first_submitted>
  <study_first_submitted_qc>June 16, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 18, 2020</study_first_posted>
  <last_update_submitted>June 18, 2020</last_update_submitted>
  <last_update_submitted_qc>June 18, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">June 22, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>gene therapy</keyword>
  <keyword>anaemia</keyword>
  <keyword>autologous hematopoeitic stem cell transplant</keyword>
  <keyword>safety</keyword>
  <keyword>bone marrow failure</keyword>
  <keyword>genetic diseases</keyword>
  <keyword>anemia</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Fanconi Syndrome</mesh_term>
    <mesh_term>Anemia</mesh_term>
    <mesh_term>Fanconi Anemia</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </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/NCT04437771
org study id: RP-L102-0116-LTFU
nct id: NCT04437771
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia
      Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial. After
      completion of the FANCOLEN-I study, eligible subjects will be followed for a total of 15
      years post gene therapy treatment. No investigational drug product will be administered
      during this study.
This long-term follow-up protocol will evaluate the long term safety and efficacy of the
      infusion of autologous CD34+ cells transduced with lentiviral vector (LV) carrying the FANCA
      gene.
observational model: Case-Only
time perspective: Prospective
measure: Monitor long term safety of patients through blood laboratory evaluations and general health status
time frame: 15 years post-drug product infusion
description: Evaluate long term safety following infusion of hematopoietic cells transduced with therapeutic lentiviral vector (LV)
measure: Long term genetic correction assessed in bone marrow and blood
time frame: 15 years post-drug product infusion
description: Determine long term persistence of therapeutic LV in hematopoietic cells in bone marrow and blood
measure: Replication competent lentivirus (RCL)
time frame: 15 years post-drug product infusion
description: Evaluate RCL in peripheral blood
measure: Insertion site analysis in blood
time frame: 15 years post-drug product infusion
description: Determine long term clonality
measure: Phenotypic correction
time frame: 15 years post-drug product infusion
description: Determine phenotypic correction of bone marrow and peripheral blood cells by resistance to DNA-damaging agents
measure: Assessment for Malignancies
time frame: 15 years post-drug product infusion
description: Monitor for incidence of hematologic malignancies and solid organ tumors
measure: Hematologic stabilization
time frame: 15 years post-drug product infusion
description: Monitor for long term stability and normalization of blood counts
arm group label: Subjects with Fanconi Anaemia Subtype A (FA-A)
description: Subjects treated with ex vivo lentiviral gene therapy product in FANCOLEN-I trial and agree to participate in this long-term follow-up (LTFU) study
intervention type: Other
intervention name: Safety and efficacy assessments
description: Long term disease and gene therapy specific safety evaluations and efficacy assessments
arm group label: Subjects with Fanconi Anaemia Subtype A (FA-A)
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
facility: 
country: Spain
responsible party type: Sponsor
mesh term: Fanconi Syndrome
mesh term: Anemia
mesh term: Fanconi Anemia
sharing ipd: No