Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia
Fanconi's Anemia
About this trial
This is an interventional treatment trial for Fanconi's Anemia focused on measuring Fanconi's anemia, aplastic anemia, hematologic disorders, rare disease
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Fanconi's anemia, complementation group C (FACC) Confirmed by diepoxybutane or mitomycin testing AND DNA analysis indicating FACC mutations Patients at least 25 kg weight No acute leukemia OR Bone marrow aspirate with greater than 10% blasts No patients who elect bone marrow transplantation --Prior/Concurrent Therapy-- At least 14 days since prior therapy for any acute viral, bacterial, or fungal infection --Patient Characteristics-- Performance status: Karnofsky 40-100% Hepatic: SGOT, SGPT, and alkaline phosphatase no greater than 5 times upper limit of normal (ULN) PT/PTT no greater than 1.5 times ULN Serum amylase no greater than 1.5 times ULN Bilirubin no greater than 2.5 mg/dL Triglyceride less than 400 mg/dL Renal: Creatinine clearance greater than 50 mL/min Cardiovascular: Normal cardiac function No ischemic heart disease that may be considered an anesthetic or operative risk Pulmonary: No lung disease that may be considered an anesthetic or operative risk Resting transcutaneous oxygen saturation greater than 90% on room air Other: HIV negative Hepatitis B surface antigen negative No underlying condition that may preclude study therapy (e.g., allergies to study reagents) No acute viral, bacterial, or fungal infection Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception