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

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified October 2003 by Office of Rare Diseases (ORD).
Recruitment status was:  Active, not recruiting
Sponsor:
ClinicalTrials.gov Identifier:
NCT00006127
First Posted: August 4, 2000
Last Update Posted: December 9, 2005
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
Office of Rare Diseases (ORD)
  Purpose

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.


Condition Intervention Phase
Fanconi's Anemia Drug: amifostine Phase 1

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 21
Study Start Date: April 2000
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

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00006127


Locations
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Dana-Farber Cancer Institute
Investigators
Study Chair: W. Nicholas Haining Dana-Farber Cancer Institute
  More Information

ClinicalTrials.gov Identifier: NCT00006127     History of Changes
Other Study ID Numbers: 199/15281
DFCI-9910170
ALZA-99-004-ii
First Submitted: August 3, 2000
First Posted: August 4, 2000
Last Update Posted: December 9, 2005
Last Verified: October 2003

Keywords provided by Office of Rare Diseases (ORD):
Fanconi's anemia
aplastic anemia
hematologic disorders
rare disease

Additional relevant MeSH terms:
Anemia
Fanconi Anemia
Fanconi Syndrome
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Amifostine
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs