Status:
UNKNOWN
FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
Lead Sponsor:
Shenzhen Geno-Immune Medical Institute
Conditions:
Fanconi Anemia
Eligibility:
All Genders
2-20 years
Phase:
NA
Brief Summary
This is a Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene. The objectives are to evaluate t...
Detailed Description
Fanconi anemia is a rare, inherited disease that is caused by a gene defect and that primarily affects an individual's bone marrow, resulting in decreased production of blood cells. The major problem ...
Eligibility Criteria
Inclusion
- Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide.
- No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection.
- Age: ≥ 4 years.
- Karnofsky: ≥ 70%.
- ANC ≥ 5×10\^8/L; PLT ≥ 2×10\^10/L.
- Hemoglobin ≥ 8g/dL.
- 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.
- Pulmonary function is normal; DLCO \> 50%.
- Written, informed consent obtained prior to any study-specific procedures.
Exclusion
- Diagnosis of active malignant disease or myelodysplastic syndrome.
- Diagnosis of myeloid leukemia.
- Pregnant or lactating females.
- Existence of an available HLA-identical related donor.
- Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive.
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
Key Trial Info
Start Date :
December 1 2017
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2021
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT03351868
Start Date
December 1 2017
End Date
December 31 2021
Last Update
September 19 2019
Active Locations (3)
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1
Capital Institute of Pediatrics affiliated Children's hospital
Beijing, Beijing Municipality, China, 100020
2
Beijing Children's Hospital
Beijing, Beijing Municipality, China
3
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China, 518000