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 is ongoing, but not recruiting participants.

First Received: February 2, 2001 Last Updated: August 23, 2006 History of Changes

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

Study Type:	Interventional
Study Design:	Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: L1 syndrome

MedlinePlus related topics: Bone Marrow Transplantation Radiation Therapy

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

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

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

Study placed in the following topic categories:

Antimetabolites
Cyclosporine
Immunologic Factors
Mycophenolic Acid
Leukocyte Disorders
Cyclosporins
Anti-Bacterial Agents
Metabolism, Inborn Errors
Antifungal Agents
Chronic Granulomatous Disease
Genetic Diseases, X-Linked
Mycophenolate mofetil
Metabolic Disorder

Metabolic Diseases
Hematologic Diseases
Fludarabine monophosphate
Granuloma
Immunosuppressive Agents
Immunologic Deficiency Syndromes
Lymphatic Diseases
Genetic Diseases, Inborn
Granulomatous Disease, Chronic
Chronic Disease
Fludarabine
Lymphoproliferative Disorders
Antirheumatic Agents

Additional relevant MeSH terms:

Antimetabolites
Anti-Infective Agents
Disease Attributes
Antimetabolites, Antineoplastic
Cyclosporine
Immunologic Factors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Physiological Effects of Drugs
Mycophenolic Acid
Leukocyte Disorders
Antibiotics, Antineoplastic
Cyclosporins
Metabolism, Inborn Errors
Pathologic Processes

Therapeutic Uses
Antifungal Agents
Genetic Diseases, X-Linked
Mycophenolate mofetil
Dermatologic Agents
Phagocyte Bactericidal Dysfunction
Metabolic Diseases
Immune System Diseases
Hematologic Diseases
Enzyme Inhibitors
Fludarabine monophosphate
Granuloma
Immunosuppressive Agents
Immunologic Deficiency Syndromes
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 02, 2009