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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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2006 by Office of Rare Diseases (ORD).
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00010361
First received: February 2, 2001
Last updated: August 23, 2006
Last verified: August 2006
History of Changes
  Purpose

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:

U.S. FDA Resources

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

Estimated Enrollment: 20
Study Start Date: November 2000
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.

  Eligibility

Ages Eligible for Study:   up to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00010361

Locations
United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
Investigators
Study Chair: Ann Woolfrey Fred Hutchinson Cancer Research Center
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00010361     History of Changes
Obsolete Identifiers: NCT00144742
Other Study ID Numbers: 199/15577, FHCRC-1475.00
Study First Received: February 2, 2001
Last Updated: August 23, 2006
Health Authority: Unspecified

Additional relevant MeSH terms:
Chronic Disease
Granulomatous Disease, Chronic
Metabolism, Inborn Errors
Granuloma
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
Lymphoproliferative Disorders
 Lymphatic Diseases
Cyclosporins
Cyclosporine
Mycophenolic Acid
Mycophenolate mofetil
Fludarabine monophosphate
Fludarabine
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents

ClinicalTrials.gov processed this record on May 23, 2013