Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 12:33 PM
Ignite Modification Date:
2025-12-24 @ 12:33 PM
NCT ID:
NCT00010361
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.
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.
Study:
NCT00010361
Study Brief:
Protocol Section:
NCT00010361