Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant
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|ClinicalTrials.gov Identifier: NCT00008450|
Recruitment Status : Completed
First Posted : January 8, 2001
Last Update Posted : July 29, 2019
|Condition or disease||Intervention/treatment||Phase|
|Adenosine Deaminase Deficiency Autosomal Recessive Disorder Immune System Disorder Purine-Nucleoside Phosphorylase Deficiency Severe Combined Immunodeficiency Severe Combined Immunodeficiency With Absence of T and B Cells X-Linked Severe Combined Immunodeficiency||Procedure: Allogeneic Bone Marrow Transplantation Drug: Cyclosporine Other: Laboratory Biomarker Analysis Drug: Mycophenolate Mofetil Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation Radiation: Total-Body Irradiation||Phase 1|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||August 11, 1997|
|Actual Primary Completion Date :||October 25, 2011|
|Actual Study Completion Date :||December 26, 2018|
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.
Procedure: Allogeneic Bone Marrow Transplantation
Undergo allogeneic bone marrow transplant
Given PO or IV
Other: Laboratory Biomarker Analysis
Drug: Mycophenolate Mofetil
Given PO or IV
Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation
Undergo nonmyeloablative allogeneic hematopoietic stem cell transplant
Radiation: Total-Body Irradiation
- Mixed hematopoietic chimerism in a population of pediatric patients with immunodeficiency diseases [ Time Frame: Up to 5 years ]It will be established whether a non-lethal conditioning regimen can successfully induce mixed hematopoietic chimerism in a population of pediatric patients with immunodeficiency diseases, without adverse effects on mortality.
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): NCT00008450
|United States, Washington|
|Fred Hutch/University of Washington Cancer Consortium|
|Seattle, Washington, United States, 98109|
|Principal Investigator:||Lauri Burroughs||Fred Hutch/University of Washington Cancer Consortium|