Last reviewed · How we verify
Hematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & Filgrastim
The goal of this study is to develop a novel approach to hematopoietic stem cell transplantation for children with Severe Combined Immunodeficiency Disease (SCID) that eliminates the use of toxic chemotherapy conditioning and maximizes the likelihood of T and B cell immune reconstitution. Rather than classic chemotherapeutic agents, the investigators will utilize a targeted stem cell mobilizer, plerixafor, in combination with alemtuzumab, a monoclonal antibody. Correlative scientific questions will include: 1) efficacy and characteristics of host stem cell mobilization; and 2) alemtuzumab pharmacokinetics in very young children.
Details
| Lead sponsor | University of California, San Francisco |
|---|---|
| Phase | Phase 2 |
| Status | TERMINATED |
| Enrolment | 7 |
| Start date | 2010-08 |
| Completion | 2013-09 |
Conditions
- Severe Combined Immunodeficiency
Interventions
- Transplant Conditioning with Mobilization Only
- Transplant Conditioning with Mobilization and Alemtuzumab
Primary outcomes
- Engraftment of Donor B-cells in Blood by STR Testing — 1 Year
Number of participants in whom donor B cells were detected in the patient's blood after HSCT.
Countries
United States