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

Primary Purpose

Fanconi Anemia

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
cyclophosphamide
cyclosporine
methotrexate
allogeneic bone marrow transplantation
nonmyeloablative allogeneic hematopoietic stem cell transplantation
Sponsored by
Fred Hutchinson Cancer Center
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fanconi Anemia focused on measuring Fanconi anemia

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Seattle Cancer Care Alliance
  • Fred Hutchinson Cancer Research Center
  • Universidade Federal do Parana

Outcomes

Primary Outcome Measures

Conditioning-related toxicity
Graft rejection

Secondary Outcome Measures

Full Information

First Posted
April 24, 2006
Last Updated
April 18, 2012
Sponsor
Fred Hutchinson Cancer Center
Collaborators
National Cancer Institute (NCI)
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1. Study Identification

Unique Protocol Identification Number
NCT00317876
Brief Title
Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia
Official Title
Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia
Study Type
Interventional

2. Study Status

Record Verification Date
April 2012
Overall Recruitment Status
Completed
Study Start Date
June 1998 (undefined)
Primary Completion Date
July 2003 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Fred Hutchinson Cancer Center
Collaborators
National Cancer Institute (NCI)

4. Oversight

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fanconi Anemia
Keywords
Fanconi anemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Masking
None (Open Label)
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
cyclophosphamide
Intervention Type
Drug
Intervention Name(s)
cyclosporine
Intervention Type
Drug
Intervention Name(s)
methotrexate
Intervention Type
Procedure
Intervention Name(s)
allogeneic bone marrow transplantation
Intervention Type
Procedure
Intervention Name(s)
nonmyeloablative allogeneic hematopoietic stem cell transplantation
Primary Outcome Measure Information:
Title
Conditioning-related toxicity
Time Frame
100 days post-transplant
Title
Graft rejection
Time Frame
100 days post-transplant

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hans-Peter Kiem, MD
Organizational Affiliation
Fred Hutchinson Cancer Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Seattle Cancer Care Alliance
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109-1023
Country
United States
Facility Name
Fred Hutchinson Cancer Research Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109-1024
Country
United States
Facility Name
Universidade Federal do Parana
City
Curitiba
State/Province
Parana
ZIP/Postal Code
80.060-000
Country
Brazil

12. IPD Sharing Statement

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

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