Gene Therapy for Fanconi Anemia, Complementation Group A (NCT04248439) | Clinical Trial Compass
Active β Not RecruitingPhase 2
Gene Therapy for Fanconi Anemia, Complementation Group A
United States5 participantsStarted 2020-07-15
Plain-language summary
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for 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
SexALL
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Inclusion criteria
β. Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent
β. Subject of the complementation group FA-A
β. Minimum age: 1 year and a minimum weight of 8 kg
β. At least 30 CD34+ cells/ΞΌL are determined in one bone marrow (BM) aspiration within 3 months prior to CD34+ cell collection OR
Exclusion criteria
β. Subjects with an available and medically eligible HLA-identical sibling donor.
β. Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities other than those reported as variant(s) of normal in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the subject commences the stem cell mobilization/collection procedures of the clinical trial.
β. Subjects 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 (β₯1 NCI CTCAE grade) in at least one blood lineage over time must be documented to enable eligibility, as should \<5% resistance of bone marrow colony forming cells (CFCs) to 10nM MMC; whenever possible potential mosaicism should also be evaluated by gene sequencing of MMC-resistant CFCs).
β. Lansky performance status β€60%.
β. Any concomitant disease or condition that, in the opinion of the Principal Investigator, renders the subject unfit to participate in the study.
What they're measuring
1
Bone Marrow (BM) Colony-Forming Cell (CFC) Mitomycin-C (MMC) resistance