Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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

Conditions
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Fanconi Anemia Complementation Group A, Fanconi Anemia

Intervention / Treatment
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* Other: Safety and efficacy assessments


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Enrolled in the FANCOLEN-I study Treated with gene therapy in the FANCOLEN-I study Able to adhere to the study visit schedule and protocol requirements Provided written informed consent and, as applicable, assent to participate Exclusion Criteria: There are no exclusion criteria for this study
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Subjects with Fanconi Anaemia Subtype A (FA-A)<br>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 | Other: Safety and efficacy assessments<br>* Long term disease and gene therapy specific safety evaluations and efficacy assessments<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Monitor long term safety of patients through blood laboratory evaluations and general health status | Evaluate long term safety following infusion of hematopoietic cells transduced with therapeutic lentiviral vector (LV) | 15 years post-drug product infusion | 
| Long term genetic correction assessed in bone marrow and blood | Determine long term persistence of therapeutic LV in hematopoietic cells in bone marrow and blood | 15 years post-drug product infusion | 
| Replication competent lentivirus (RCL) | Evaluate RCL in peripheral blood | 15 years post-drug product infusion | 
| Insertion site analysis in blood | Determine long term clonality | 15 years post-drug product infusion | 
| Phenotypic correction | Determine phenotypic correction of bone marrow and peripheral blood cells by resistance to DNA-damaging agents | 15 years post-drug product infusion | 
| Assessment for Malignancies | Monitor for incidence of hematologic malignancies and solid organ tumors | 15 years post-drug product infusion | 
| Hematologic stabilization | Monitor for long term stability and normalization of blood counts | 15 years post-drug product infusion | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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safety, genetic diseases, anaemia, gene therapy, autologous hematopoeitic stem cell transplant, bone marrow failure, anemia