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Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders

Primary Purpose

Metabolism, Inborn Errors, Granulomatous Disease, Chronic

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
cyclosporine
fludarabine
mycophenolate mofetil
Sponsored by
Fred Hutchinson Cancer Center
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Metabolism, Inborn Errors

Eligibility Criteria

undefined - 55 Years (Child, Adult)All SexesDoes not accept healthy volunteers

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Inherited disorders treatable with allogeneic peripheral blood or bone marrow transplantation At high risk for regimen related toxicity with a conventional transplant No severe CNS involvement of disease, defined by IQ score less than 70 HLA matched donor Sibling donors must be a confirmed match at HLA-A, B, and DRB1 Other related and non-related donors must be matched at HLA-A, B, C, DRB1, and DQB1 A donor homozygous for one allele only at HLA-A, B, C, DRB1, or DQB1 allowed (1 antigen mismatch for graft-versus-host disease, 0 antigen mismatch for graft-rejection) --Prior/Concurrent Therapy-- No concurrent growth factors with mycophenolate mofetil --Patient Characteristics-- Age: Under 55 Performance status: Not specified Life expectancy: At least 100 days Hematopoietic: Not specified Hepatic: No evidence of synthetic dysfunction No severe cirrhosis Renal: Not specified Cardiovascular: LVEF at least 30% No poorly controlled hypertension on multiple antihypertensives Other: No organ dysfunction that would preclude survival Not pregnant or nursing Fertile patients must use effective contraception during and for 12 months following study

Sites / Locations

  • Fred Hutchinson Cancer Research Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
February 2, 2001
Last Updated
December 8, 2014
Sponsor
Fred Hutchinson Cancer Center
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1. Study Identification

Unique Protocol Identification Number
NCT00010361
Brief Title
Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
Study Type
Interventional

2. Study Status

Record Verification Date
August 2006
Overall Recruitment Status
Completed
Study Start Date
November 2000 (undefined)
Primary Completion Date
April 2007 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Fred Hutchinson Cancer Center

4. Oversight

5. Study Description

Brief Summary
OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by allogeneic peripheral blood stem cell or bone marrow transplantation in combination with cyclosporine and mycophenolate mofetil for establishing mixed chimerism in patients with inherited disorders. II. Determine whether this regimen can establish mixed chimerism in these patients. III. Determine whether mixed chimerism is sufficient to reverse disease symptoms in these patients. IV. Determine the safety of donor lymphocyte infusions to eliminate persistent disease in these patients with mixed chimerism.
Detailed Description
PROTOCOL OUTLINE: Patients receive fludarabine IV over 2 hours on days -4 to -2 followed by total body irradiation and peripheral blood stem cell or bone marrow transplantation on day 0. Patients also receive oral or IV cyclosporine 2-3 times daily on days -3 to 50 (related donor) or 100 (unrelated donor) and oral mycophenolate mofetil twice daily on days 0 to 28 (related donor) or 40 (unrelated donor). Patients may also receive donor lymphocyte infusion for continued treatment of symptoms in the event of mixed chimerism and in the absence of graft-versus-host disease. Patients are followed weekly for 1 month, monthly for 2 years, and then annually thereafter.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Metabolism, Inborn Errors, Granulomatous Disease, Chronic

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Enrollment
20 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
cyclosporine
Intervention Type
Drug
Intervention Name(s)
fludarabine
Intervention Type
Drug
Intervention Name(s)
mycophenolate mofetil

10. Eligibility

Sex
All
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Inherited disorders treatable with allogeneic peripheral blood or bone marrow transplantation At high risk for regimen related toxicity with a conventional transplant No severe CNS involvement of disease, defined by IQ score less than 70 HLA matched donor Sibling donors must be a confirmed match at HLA-A, B, and DRB1 Other related and non-related donors must be matched at HLA-A, B, C, DRB1, and DQB1 A donor homozygous for one allele only at HLA-A, B, C, DRB1, or DQB1 allowed (1 antigen mismatch for graft-versus-host disease, 0 antigen mismatch for graft-rejection) --Prior/Concurrent Therapy-- No concurrent growth factors with mycophenolate mofetil --Patient Characteristics-- Age: Under 55 Performance status: Not specified Life expectancy: At least 100 days Hematopoietic: Not specified Hepatic: No evidence of synthetic dysfunction No severe cirrhosis Renal: Not specified Cardiovascular: LVEF at least 30% No poorly controlled hypertension on multiple antihypertensives Other: No organ dysfunction that would preclude survival Not pregnant or nursing Fertile patients must use effective contraception during and for 12 months following study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ann Woolfrey
Organizational Affiliation
Fred Hutchinson Cancer Center
Official's Role
Study Chair
Facility Information:
Facility Name
Fred Hutchinson Cancer Research Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders

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