NCT00010361

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

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 20 (planned)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 55 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Metabolism, Inborn Errors
• Granulomatous Disease, Chronic

Interventions

Type	Name	Description
DRUG	cyclosporine
DRUG	fludarabine
DRUG	mycophenolate mofetil

Timeline

• Start date: 2000-11-01
• Primary completion: 2007-04-01
• First posted: 2001-02-02
• Last updated: 2014-12-09

Locations

1 site across 1 country: United States