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

NCT ID: NCT00006127

Last Updated: 2005-06-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2000-04-30

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Fanconi's Anemia

Keywords

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Fanconi's anemia aplastic anemia hematologic disorders rare disease

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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amifostine

Intervention Type DRUG

Eligibility Criteria

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Inclusion 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
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Dana-Farber Cancer Institute

OTHER

Sponsor Role lead

Principal Investigators

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W. Nicholas Haining

Role: STUDY_CHAIR

Dana-Farber Cancer Institute

Locations

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Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

Countries

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United States

Other Identifiers

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DFCI-9910170

Identifier Type: -

Identifier Source: secondary_id

ALZA-99-004-ii

Identifier Type: -

Identifier Source: secondary_id

199/15281

Identifier Type: -

Identifier Source: org_study_id