Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant
|Immune System Disorder Severe Combined Immunodeficiency||Drug: cyclosporine Drug: mycophenolate mofetil Radiation: total-body irradiation Procedure: allogeneic bone marrow transplantation Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation Other: laboratory biomarker analysis||Phase 1|
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||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|
- 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 [ Time Frame: Up to 5 years ]
- Extent of immune deficiency and tempo of immune reconstitution of lymphoid subsets T (CD4+, CD8+, CD3+), B (CD19+, CD20+), and NK (CD56+) as assessed by flow cytometry [ Time Frame: Up to 5 years ]
|Study Start Date:||August 1997|
|Primary Completion Date:||October 2011 (Final data collection date for primary outcome measure)|
Experimental: Treatment (cyclosporine, mycophenolate mofetil, transplant)
Patients receive cyclosporine PO or 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.
Given PO or IV
Other Names:Drug: mycophenolate mofetil
Given PO or IV
Other Names:Radiation: total-body irradiation
Other Name: TBIProcedure: allogeneic bone marrow transplantation
Undergo allogeneic bone marrow transplant
Other Names:Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation
Undergo nonmyeloablative allogeneic hematopoietic stem cell transplantOther: laboratory biomarker analysis
I. To safely establish partial lymphoid chimerism (1-95% donor cluster of differentiation [CD]3+ 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 yearly for 5 years.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00008450
|United States, Washington|
|Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium|
|Seattle, Washington, United States, 98109|
|Principal Investigator:||Lauri Burroughs||Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium|