Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
This trial is active, not recruiting.
|Sponsor||Dana-Farber Cancer Institute|
|Start date||April 2000|
|Trial size||21 participants|
|Trial identifier||NCT00006127, 199/15281, ALZA-99-004-ii, DFCI-9910170|
I. Evaluate the toxicity of amifostine in patients with bone marrow failure related to Fanconi's anemia.
II. Determine the efficacy of this treatment regimen in this patient population.
III. Evaluate the effect of this treatment regimen on bone marrow progenitor cell proliferation and peripheral blood mononuclear cell apoptosis in these patients.
Male or female participants at least 2 years old.
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Diagnosis of Fanconi's anemia by conventional diepoxybutane or mitomycin sensitivity assays, or molecular testing Bone marrow failure verified on at least 3 occasions in the preceding 3 months by any one of the following: hemoglobin less than 8 g/dL; absolute neutrophil count less than 1,000/mm3; platelet count less than 30,000/mm3; symptomatic bone marrow failure (e.g., exercise limitation from anemia or spontaneous bleeding from thrombocytopenia) Evidence consistent with myelodysplastic syndrome allowed if less than 5% blasts on bone marrow aspiration; clonality on bone marrow cytogenetic analysis OR morphological changes on bone marrow aspirate Refusal of or unsuccessful with prior conventional therapies --Prior/Concurrent Therapy-- Biologic therapy: No prior bone marrow transplantation; no concurrent hematopoietic growth factors Endocrine therapy: No concurrent androgens --Patient Characteristics-- Hepatic: Bilirubin no greater than 2 times normal AST no greater than 3 times normal ALT no greater than 6 times normal Renal: Creatinine no greater than 2 times normal Other: No prior malignancy; no active bacterial, viral, or fungal infection requiring therapy other than prophylaxis; not pregnant; negative pregnancy test
|Description||PROTOCOL OUTLINE: This is a dose escalation study. Patients receive amifostine IV over 3-5 minutes three times a week for three weeks. Cohorts of 3 patients receive one of three dose levels of amifostine. The maximum tolerated dose is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity. Patients are followed weekly for 3 weeks.|
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