Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim
Status:
Terminated
Trial end date:
2013-09-01
Target enrollment:
Participant gender:
Summary
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.