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Conditioning SCID Infants Diagnosed Early
Condition: SCID · Sponsor: Center for International Blood and Marrow Transplant Research
About this study
The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants. The study will test if patients receiving transplant using either a low dose busulfan or a medium dose busulfan will have immune recovery of both T and B cells, measured by the ability to respond to immunizations after transplant. The exact regimen depends on the subtype of SCID the patient has. Donors used for transplant must be unrelated or half-matched related (haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T cells removed, using a newer, experimental approach of a well-established technology. Once the stem cell transplant is completed, patients will be followed for 3 years. Approximately 9-18 months after the transplant, vaccinations will be administered, and a blood test measuring whether your child's body has responded to the vaccine will be collected.
This description comes directly from the study's public registry record.
Talk to the study team
No contact information is available for this study as per the public registry record.
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Locations (42)
| Univeristy of Alabama at Birmingham | Birmingham, Alabama, United States | — |
| Mayo Clinic Arizona and Phoenix Children's Hospital | Phoenix, Arizona, United States | — |
| Children's Hospital Los Angeles | Los Angeles, California, United States | — |
| UCLA Center for Health Sciences | Los Angeles, California, United States | — |
| Rady Children's Hospital, San Diego | San Diego, California, United States | — |
| University of California San Francisco Medical Center - Peds | San Francisco, California, United States | — |
| University of Colorado - Children's Hospital | Aurora, Colorado, United States | — |
| Children's National Medical Center | Washington D.C., District of Columbia, United States | — |
| Shands HealthCare & University of Florida | Gainesville, Florida, United States | — |
| University of Miami/Jackson Memorial Hospital | Miami, Florida, United States | — |
| All Children's Hospital | St. Petersburg, Florida, United States | — |
| Children's Healthcare of Atlanta at Egleston | Atlanta, Georgia, United States | — |
+ 30 more locations — full list on the registry record.
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