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
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|ClinicalTrials.gov Identifier: NCT00010361|
Recruitment Status : Completed
First Posted : February 2, 2001
Last Update Posted : December 9, 2014
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 or disease||Intervention/treatment||Phase|
|Metabolism, Inborn Errors Granulomatous Disease, Chronic||Drug: cyclosporine Drug: fludarabine Drug: mycophenolate mofetil||Not Applicable|
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.
|Study Type :||Interventional (Clinical Trial)|
|Enrollment :||20 participants|
|Study Start Date :||November 2000|
|Actual Primary Completion Date :||April 2007|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00010361
|United States, Washington|
|Fred Hutchinson Cancer Research Center|
|Seattle, Washington, United States, 98109|
|Study Chair:||Ann Woolfrey||Fred Hutchinson Cancer Research Center|