Status:
TERMINATED
Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim
Lead Sponsor:
University of California, San Francisco
Conditions:
Severe Combined Immunodeficiency
Eligibility:
All Genders
Up to 3 years
Phase:
PHASE2
Brief 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 chem...
Detailed Description
The goal of this study is to develop an approach to hematopoietic stem cell transplantation for children with Severe Combined Immunodeficiency Disease (SCID) that eliminates the use of toxic chemother...
Eligibility Criteria
Inclusion
- Patients with classic SCID phenotype (\<400 CD3/ul or maternally engrafted and \<10% of normal PHA lymphoproliferative response). Genotypic identification is preferable, but not required.
- Patients must have an acceptable stem cell donor (HLA matched relative, 9 or 10/10 HLA-matched unrelated, or haplocompatible relative).
Exclusion
- Patients with "leaky" SCID syndromes, Omenn's Syndrome, reticular dysgenesis, ADA deficiency
- Lansky score \<60%
- Patient with expected survival \<4 weeks (including disseminated CMV infection involving lungs and/or CNS)
Key Trial Info
Start Date :
August 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2013
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT01182675
Start Date
August 1 2010
End Date
September 1 2013
Last Update
July 31 2018
Active Locations (1)
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1
UCSF Benioff Children's Hospital
San Francisco, California, United States, 94143