Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A (NCT04069533) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
Spain, United Kingdom7 participantsStarted 2019-11-28
Plain-language summary
This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).
Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.
Who can participate
Age range1 Year – 17 Years
SexALL
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Inclusion criteria
✓. Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of diepoxybutane (DEB) or similar DNA-crosslinking agent
✓. Patient of the complementation group FA-A
✓. Minimum age: 1 year and minimum weight of 8 kg.
✓. Maximum age: 17 years
✓. At least 30 CD34+ cells/µL are determined in one BM aspiration within 3 months prior to the CD34+ cell collection.
✓. Provide informed consent in accordance with current legislation
✓. Women of childbearing age must have a negative urine pregnancy test at the baseline visit, and accept the use of an effective contraception method during participation in the trial
Exclusion criteria
✕. Patients with an available and medically eligible human leukocyte antigen (HLA)-identical sibling donor
✕. Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities other than those predictive of these conditions in bone marrow (BM) aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the patient enters the clinical trial
✕. Patients with somatic mosaicism associated with stable or improved counts in all PB cell lineages (If T-lymphocyte chromosomal fragility analysis indicates potential mosaicism, a medically significant decrease in at least one blood lineage over time must be documented to enable eligibility)
What they're measuring
1
Phenotypic correction of bone marrow colony forming units after infusion of RP-L102