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

Primary Purpose

Fanconi's Anemia

Status
Unknown status
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
amifostine
Sponsored by
Dana-Farber Cancer Institute
About
Eligibility
Locations
Outcomes
Full info

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

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Dana-Farber Cancer Institute

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
August 3, 2000
Last Updated
June 23, 2005
Sponsor
Dana-Farber Cancer Institute
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1. Study Identification

Unique Protocol Identification Number
NCT00006127
Brief Title
Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia
Study Type
Interventional

2. Study Status

Record Verification Date
October 2003
Overall Recruitment Status
Unknown status
Study Start Date
April 2000 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Dana-Farber Cancer Institute

4. Oversight

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fanconi's Anemia
Keywords
Fanconi's anemia, aplastic anemia, hematologic disorders, rare disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Enrollment
21 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
amifostine

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
W. Nicholas Haining
Organizational Affiliation
Dana-Farber Cancer Institute
Official's Role
Study Chair
Facility Information:
Facility Name
Dana-Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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