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

This study is ongoing, but not recruiting participants.

Sponsored by: Dana-Farber Cancer Institute
Information provided by: Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier: NCT00006127
  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 I

MedlinePlus related topics:   Anemia   

ChemIDplus related topics:   Amifostine   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment

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
Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
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

Please refer to this study by its ClinicalTrials.gov identifier: 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


Study ID Numbers:   199/15281, DFCI-9910170, ALZA-99-004-ii
First Received:   August 3, 2000
Last Updated:   June 23, 2005
ClinicalTrials.gov Identifier:   NCT00006127
Health Authority:   Unspecified

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

Study placed in the following topic categories:
Metabolic Diseases
Amifostine
Hematologic Diseases
Fanconi Anemia
Anemia
Rare Diseases
Pancytopenia
Genetic Diseases, Inborn
Fanconi's anemia
Anemia, Aplastic
Bone Marrow Diseases
Aplastic anemia
Metabolic disorder

Additional relevant MeSH terms:
Radiation-Protective Agents
Anemia, Hypoplastic, Congenital
Physiological Effects of Drugs
DNA Repair-Deficiency Disorders
Protective Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 03, 2008




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