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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00010361
Recruitment Status : Completed
First Posted : February 2, 2001
Last Update Posted : December 9, 2014
Information provided by:
Office of Rare Diseases (ORD)

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.

Condition or disease Intervention/treatment Phase
Metabolism, Inborn Errors Granulomatous Disease, Chronic Drug: cyclosporine Drug: fludarabine Drug: mycophenolate mofetil Not Applicable

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.

Study Type : Interventional  (Clinical Trial)
Enrollment : 20 participants
Primary Purpose: Treatment
Study Start Date : November 2000
Actual Primary Completion Date : April 2007

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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): 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 Identifier: NCT00010361     History of Changes
Obsolete Identifiers: NCT00144742
Other Study ID Numbers: 199/15577
First Posted: February 2, 2001    Key Record Dates
Last Update Posted: December 9, 2014
Last Verified: August 2006

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