Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim

Stopped early · Phase 2

Conditions studied: Severe Combined Immunodeficiency

In brief

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.

Key facts

Study ID

NCT01182675

Run by

University of California, San Francisco

People needed

7

Starts

2010-08-01

Expected to finish

2013-09-01

Last updated by the study team

2018-07-31

Who can join

Age: any, up to 3. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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).

You may not qualify if…

• 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)

Where it is running

• UCSF Benioff Children's Hospital — San Francisco, California, United States

Full record on ClinicalTrials.gov

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