Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia
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Purpose
RATIONALE: Giving low doses of chemotherapy, such as cyclophosphamide, before a donor bone marrow transplant helps stop the growth of abnormal cells. It also stops the patient's immune system from rejecting the donor's bone marrow. The donated bone marrow stem cells may replace the patient's immune system and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine and methotrexate before or after transplant may stop this from happening.
PURPOSE: This phase I trial is studying the side effects and best dose of cyclophosphamide in treating patients who are undergoing a donor bone marrow transplant for Fanconi's anemia.
| Condition | Intervention | Phase |
|---|---|---|
|
Fanconi Anemia |
Drug: cyclophosphamide Drug: cyclosporine Drug: methotrexate Procedure: allogeneic bone marrow transplantation Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia |
- Conditioning-related toxicity [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
- Graft rejection [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: No ]
| Enrollment: | 25 |
| Study Start Date: | June 1998 |
| Primary Completion Date: | July 2003 (Final data collection date for primary outcome measure) |
OBJECTIVES:
- Decrease the conditioning-related toxicity of cyclophosphamide without decreasing the engraftment rate to < 90% in patients undergoing allogeneic bone marrow transplantation for Fanconi's anemia.
OUTLINE: This is a multicenter, dose-finding study of cyclophosphamide.
- Nonmyeloablative conditioning regimen: Patients receive cyclophosphamide IV on days -5 to -2.
Cohorts of 5-10 patients receive decreasing doses of cyclophosphamide until the optimal dose (OD) is determined. The OD is defined as the dose at which ≥ 4 of 5 patients achieve engraftment and < 1 of 10 patients experiences dose-limiting toxicity.
- Allogeneic bone marrow transplantation (BMT): Patients undergo allogeneic BMT on day 0.
- Graft-vs-host-disease (GVHD) prophylaxis: Patients receive cyclosporine orally or IV twice daily beginning on day -1 and continuing until day 49, followed by a taper on days 50-180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11.
After completion of study treatment, patients are followed periodically for 5 years.
PROJECTED ACCRUAL: A total of 27 patients will be accrued for this study.
Eligibility| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Diagnosis of Fanconi's anemia by chromosome fragility with a diepoxybutane (DEB) or mitomycin C test
- Hemoglobin ≤ 8.0 g/dL, absolute granulocyte count ≤ 1,000/mm^3, or platelet count ≤ 50,000/mm^3
- No refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute leukemia
- HLA-identical related donor available
PATIENT CHARACTERISTICS:
- Glomerular filtration rate ≥ 30% predicted for age
- No liver disease (e.g., active hepatitis or moderate to severe portal fibrosis/cirrhosis by biopsy)
- No symptomatic cardiac insufficiency or symptomatic arrhythmia
- No other diseases that would severely limit the probability of survival
- No HIV seropositivity
- Not pregnant or nursing
- Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
- Not specified
Contacts and Locations| United States, Washington | |
| Fred Hutchinson Cancer Research Center | |
| Seattle, Washington, United States, 98109-1024 | |
| Seattle Cancer Care Alliance | |
| Seattle, Washington, United States, 98109-1023 | |
| Brazil | |
| Universidade Federal do Parana | |
| Curitiba, Parana, Brazil, 80.060-000 | |
| Principal Investigator: | Hans-Peter Kiem, MD | Fred Hutchinson Cancer Research Center |
More Information
Additional Information:
No publications provided
| Responsible Party: | Hans-Peter Kiem, MD, Fred Hutchinson Cancer Research Center |
| ClinicalTrials.gov Identifier: | NCT00317876 History of Changes |
| Other Study ID Numbers: | 1288.00, FHCRC-1288.00, CDR0000481264 |
| Study First Received: | April 24, 2006 |
| Last Updated: | April 18, 2012 |
| Health Authority: | United States: Federal Government |
Keywords provided by Fred Hutchinson Cancer Research Center:
|
Fanconi anemia |
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 Cyclophosphamide |
Cyclosporins Cyclosporine Methotrexate Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Antirheumatic Agents Therapeutic Uses Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Enzyme Inhibitors |
ClinicalTrials.gov processed this record on May 21, 2013