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N/A
N/A
55 Years
Open (Enrolling)
Both
Metabolism, Inborn Errors, Granulomatous Disease, Chronic

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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 CenterSeattle, Washington  98109