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

NCT00006127 | Unknown Phase 1

• 3 other identifiers: 199/15281DFCI-9910170ALZA-99-004-ii
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Fanconi's Anemia

Keywords

Fanconi's anemia; aplastic anemia; hematologic disorders; rare disease

Interventions

amifostine DRUG

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead

Study Sites (1)

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Fanconi Anemia; Anemia, Aplastic; Hematologic Diseases; Rare Diseases

Interventions

Amifostine

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, Congenital; Anemia; Hemic and Lymphatic Diseases; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Organothiophosphates; Organophosphates; Organophosphorus Compounds; Organic Chemicals; Organothiophosphorus Compounds; Sulfur Compounds

Study Officials

• W. Nicholas Haining

  Dana-Farber Cancer Institute

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: August 3, 2000
• First Posted: August 4, 2000
• Study Start: April 1, 2000
• Last Updated: June 24, 2005

Record last verified: 2003-10

Locations

US (1)