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

NCT00010361 | Completed

• 2 other identifiers: 199/15577FHCRC-1475.00
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Metabolism, Inborn Errors; Granulomatous Disease, Chronic

Interventions

cyclosporine DRUG

fludarabine DRUG

mycophenolate mofetil DRUG

Eligibility Criteria

• Age: Up to 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Fred Hutchinson Cancer Center: lead

Study Sites (1)

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Metabolism, Inborn Errors; Granulomatous Disease, Chronic

Interventions

Cyclosporine; fludarabine; Mycophenolic Acid

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Phagocyte Bactericidal Dysfunction; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, X-Linked; Immunologic Deficiency Syndromes; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins; Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• Ann Woolfrey

  Fred Hutchinson Cancer Center

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: February 2, 2001
• First Posted: February 2, 2001
• Study Start: November 1, 2000
• Primary Completion: April 1, 2007
• Last Updated: December 9, 2014

Record last verified: 2006-08

Locations

US (1)