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 - Full Text View

Sponsored by:	Fred Hutchinson Cancer Research Center
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00010361

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

Genetics Home Reference related topics: L1 syndrome

MedlinePlus related topics: Bone Marrow Transplantation

Drug Information available for: Fludarabine Fludarabine monophosphate Cyclosporin Cyclosporine Mycophenolate Mofetil Mycophenolate mofetil hydrochloride

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment

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

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

Study placed in the following topic categories:

Cyclosporine
Metabolic Diseases
Clotrimazole
Hematologic Diseases
Miconazole
Tioconazole
Mycophenolic Acid
Leukocyte Disorders
Fludarabine monophosphate
Granuloma
Cyclosporins
Immunologic Deficiency Syndromes

Metabolism, Inborn Errors
Lymphatic Diseases
Genetic Diseases, Inborn
Granulomatous Disease, Chronic
Mycophenolate mofetil
Genetic Diseases, X-Linked
Chronic Disease
Fludarabine
Metabolic disorder
Lymphoproliferative Disorders
Chronic granulomatous disease

Additional relevant MeSH terms:

Antimetabolites
Phagocyte Bactericidal Dysfunction
Anti-Infective Agents
Disease Attributes
Antimetabolites, Antineoplastic
Immune System Diseases
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antineoplastic Agents
Physiological Effects of Drugs

Enzyme Inhibitors
Antibiotics, Antineoplastic
Immunosuppressive Agents
Pharmacologic Actions
Pathologic Processes
Antifungal Agents
Therapeutic Uses
Antirheumatic Agents
Dermatologic Agents

ClinicalTrials.gov processed this record on January 16, 2009