This study examines a gene therapy in which the FANCA gene is delivered into the patient's own stem cells using a self-inactivating lentiviral vector, for patients with FANCA-type Fanconi anemia. It enrolls patients aged 4 and older whose FANCA-type Fanconi anemia is confirmed by DNA sequencing. The trial is a non-phase study and is currently recruiting.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide. 2. No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection. 3. Age: ≥ 4 years. 4. Karnofsky: ≥ 70%. 5. ANC ≥ 5×10\^8/L; PLT ≥ 2×10\^10/L. 6. Hemoglobin ≥ 8g/dL. 7. Proper renal and hepatic functions (ULN denotes "upper limit of normal range") with * serum creatinine ≤ 1.5×ULN; * serum bilirubin ≤ 3×ULN; * AST/ALT ≤ 5×ULN. 8. Pulmonary function is normal; DLCO \> 50%. 9. Written, informed consent obtained prior to any study-specific procedures. Exclusion Criteria: 1. Diagnosis of active malignant disease or myelodysplastic syndrome. 2. Diagnosis of myeloid leukemia. 3. Pregnant or lactating females. 4. Existence of an available HLA-identical related donor. 5. Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive. 6. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.