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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

This study has been completed.
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: February 2, 2001
Last updated: December 8, 2014
Last verified: August 2006

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.

Condition Intervention
Metabolism, Inborn Errors
Granulomatous Disease, Chronic
Drug: cyclosporine
Drug: fludarabine
Drug: mycophenolate mofetil

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 20
Study Start Date: November 2000
Primary Completion Date: April 2007 (Final data collection date for primary outcome measure)
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.


Ages Eligible for Study:   up to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00010361

United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
Study Chair: Ann Woolfrey Fred Hutchinson Cancer Research Center
  More Information Identifier: NCT00010361     History of Changes
Obsolete Identifiers: NCT00144742
Other Study ID Numbers: 199/15577
Study First Received: February 2, 2001
Last Updated: December 8, 2014

Additional relevant MeSH terms:
Chronic Disease
Granulomatous Disease, Chronic
Metabolism, Inborn Errors
Disease Attributes
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases
Metabolic Diseases
Fludarabine phosphate
Mycophenolic Acid
Mycophenolate mofetil
Antineoplastic Agents
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Enzyme Inhibitors
Antibiotics, Antineoplastic
Antifungal Agents
Anti-Infective Agents processed this record on March 30, 2017