Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia - Full Text View

Sponsors and Collaborators:	Fred Hutchinson Cancer Research Center National Cancer Institute (NCI)
Information provided by:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT00317876

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 I

MedlinePlus related topics: Anemia Bone Marrow Transplantation Cancer

Drug Information available for: Cyclophosphamide Methotrexate Cyclosporin Cyclosporine

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label
Official Title:	Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:

Conditioning-related toxicity [ Designated as safety issue: Yes ]
Graft rejection [ Designated as safety issue: No ]

Estimated Enrollment:	27
Study Start Date:	June 1998
Estimated Primary Completion Date:	June 2009 (Final data collection date for primary outcome measure)

Detailed Description:

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

Criteria

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00317876

Locations

United States, Washington
Fred Hutchinson Cancer Research Center	Recruiting
Seattle, Washington, United States, 98109-1024
Contact: Hans-Peter Kiem, MD 206-667-4425
Seattle Cancer Care Alliance	Recruiting
Seattle, Washington, United States, 98109-1023
Contact: Clinical Trials Office - Seattle Cancer Care Alliance 800-804-8824
Brazil, Parana
Universidade Federal do Parana	Recruiting
Curitiba, Parana, Brazil, 80.060-000
Contact: Ricardo Pasquini, MD 55-41-3360-1800

Sponsors and Collaborators

Fred Hutchinson Cancer Research Center

National Cancer Institute (NCI)

Investigators

Principal Investigator:

Hans-Peter Kiem, MD

Fred Hutchinson Cancer Research Center

More Information

Clinical trial summary from the National Cancer Institute's PDQ® database This link exits the ClinicalTrials.gov site

Study ID Numbers:	CDR0000481264, FHCRC-1288.00
Study First Received:	April 24, 2006
Last Updated:	December 31, 2008
ClinicalTrials.gov Identifier:	NCT00317876
Health Authority:	Unspecified

Keywords provided by National Cancer Institute (NCI):

Fanconi anemia

Study placed in the following topic categories:

Metabolic Diseases
Cyclosporine
Clotrimazole
Hematologic Diseases
Miconazole
Fanconi Anemia
Tioconazole
Anemia
Cyclophosphamide
Cyclosporins

Folic Acid
Signs and Symptoms
Genetic Diseases, Inborn
Fanconi's anemia
Anemia, Aplastic
Methotrexate
Bone Marrow Diseases
Aplastic anemia
Metabolic disorder

Additional relevant MeSH terms:

Antimetabolites
Anti-Infective Agents
Antimetabolites, Antineoplastic
Immunologic Factors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
DNA Repair-Deficiency Disorders
Physiological Effects of Drugs
Enzyme Inhibitors
Reproductive Control Agents
Folic Acid Antagonists
Abortifacient Agents, Nonsteroidal

Immunosuppressive Agents
Pharmacologic Actions
Anemia, Hypoplastic, Congenital
Antifungal Agents
Therapeutic Uses
Abortifacient Agents
Myeloablative Agonists
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Dermatologic Agents
Alkylating Agents
Nucleic Acid Synthesis Inhibitors

ClinicalTrials.gov processed this record on January 16, 2009