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About
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.
Full description
This is a pediatric open-label Phase II clinical trial to assess the efficacy of a hematopoietic gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in pediatric subjects with FA-A.
Enriched CD34+ hematopoietic stem cells will be transduced ex vivo with the therapeutic lentiviral vector and infused via intravenous infusion following transduction without any prior conditioning.
Enrollment
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Inclusion criteria
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)
Lansky performance index ≤ 60%
Any concomitant disease or condition that, in the opinion of the Principal Investigator, deems the patient unfit to participate in the trial
Pre-existing sensory or motor impairment >/= grade 2 according to the criteria of the National Cancer Institute (NCI)
Pregnant or breastfeeding women
Hepatic dysfunction as defined by either:
Renal dysfunction requiring either hemodialysis or peritoneal dialysis
Pulmonary dysfunction as defined by either:
Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years
Subject is receiving androgens (i.e. danazol, oxymetholone)
Subject is receiving other investigational therapy for treatment/prevention of FA-associated bone marrow failure
Primary purpose
Allocation
Interventional model
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7 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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