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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified October 2003 by Office of Rare Diseases (ORD).
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00006127
First received: August 3, 2000
Last updated: June 23, 2005
Last verified: October 2003
  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

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

No publications provided

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

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
Kidney Diseases
Urologic Diseases
Renal Tubular Transport, Inborn Errors
Metabolism, Inborn Errors
Amifostine
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 16, 2014