Overview

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

Status:
Unknown status

Trial end date:
1969-12-31

Target enrollment:
0

Participant gender:
All

Summary

OBJECTIVES: 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.

Phase:

Phase 1

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Dana-Farber Cancer Institute

Treatments:

Amifostine

Criteria

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