Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
Information source: Office of Rare Diseases (ORD)
Information obtained from ClinicalTrials.gov on May 08, 2007
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Fanconi's Anemia
Intervention: amifostine (Drug)
Phase: Phase 1
Status: No longer recruiting
Sponsored by: Dana-Farber Cancer Institute Official(s) and/or principal investigator(s):
W. Nicholas Haining, Study Chair, Affiliation: Dana-Farber Cancer Institute
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.
Clinical Details
Study design: Interventional, Treatment
Detailed 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.
Eligibility
Minimum age: 2 Years.
Gender(s): Both.
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
Locations and Contacts
Dana-Farber Cancer Institute, Boston, Massachusetts 02115, United States
Additional Information
Starting date:
April 2000
Last updated: June 23, 2005
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