Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil
I. To safely establish partial lymphoid chimerism (1-95% donor CD3+ cells) using a
non-lethal conditioning regimen in patients with Severe Combined Immunodeficiency Syndrome.
II. To define the kinetics of immune reconstitution following a non-lethal conditioning
regimen in patients with immunodeficiency diseases.
Patients receive cyclosporine orally (PO) or intravenously (IV) on days -3 to 100 followed
by a taper until day 180 and mycophenolate mofetil PO or IV on days 0-40 with a taper until
day 96 in the absence of unacceptable toxicity. Unrelated donor recipients also undergo TBI
on day 0. Patients undergo bone marrow transplant on day 0.
After completion of study treatment, patients are followed up at 6 months and then every
year for 5 years.
Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Extent of chimerism in specific subpopulations by assessing T (CD3+3), B (CD19/20+), NK (CD56+), and myeloid (CD33+/13+) of bone marrow aspirate and peripheral blood
Up to 5 years
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
United States: Food and Drug Administration
|Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium||Seattle, Washington 98109|