N/A
55 Years
Both
Metabolism, Inborn Errors, Granulomatous Disease, Chronic
Trial Information
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.
Inclusion 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
Type of Study:
Interventional
Study Design:
Primary Purpose: Treatment
Principal Investigator
Ann Woolfrey
Investigator Role:
Study Chair
Investigator Affiliation:
Fred Hutchinson Cancer Research Center
Authority:
Unspecified
Study ID:
199/15577
NCT ID:
NCT00010361
Start Date:
November 2000
Completion Date:
Related Keywords:
- Metabolism, Inborn Errors
- Granulomatous Disease, Chronic
- Chronic Disease
- Granulomatous Disease, Chronic
- Metabolism, Inborn Errors
- Granuloma
Name | Location |
|---|---|
| Fred Hutchinson Cancer Research Center | Seattle, Washington 98109 |
