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Hematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & Filgrastim

Phase 2
3 Years
Not Enrolling
Severe Combined Immunodeficiency

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Trial Information

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 an 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. SCID is a rare primary immunodeficiency disease in which there are multiple
genotypes and phenotypes, and depending on various factors including the presence of B cell
and NK cells, and the presence of maternal cells in the patient's circulation, there are
numerous ways to approach a transplant. The major issues that must be addressed in any
approach to transplantation for SCID are graft rejection and T and B cell immune
reconstitution. Depending on the specific SCID diagnosis, the phenotype, and the presence of
maternal engraftment at diagnosis, we will evaluate two transplant approaches that will
attempt to optimize the engraftment of donor HSC and the likelihood of T and B cell
reconstitution while eliminating the use of toxic chemotherapy conditioning.

1. Primary Objective: To determine if the administration of plerixafor & filgrastim
(G-CSF) prior to stem cell infusion results in increased donor stem cell occupancy of
available bone marrow niches and B-cell engraftment in patients with SCID.

2. Secondary Objectives:

i. To determine if NK cell depletion with Alemtuzumab will overcome NK-mediated graft
resistance in haplocompatible transplants for NK+ SCID.

ii. To determine the optimal dosing of Alemtuzumab in very young children. iii. To determine
the immunophenotypic characteristics of CD34+ cells mobilized and engrafted in patients
receiving plerixafor & filgrastim prior to HCT.

iv. To determine the thymic output, as measured by T-cell receptor excision circles, in
patients receiving haplocompatible transplants & boosts.

Inclusion Criteria:

- 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 Criteria:

- Patients with "leaky" SCID syndromes, Omenn's Syndrome, reticular dysgenesis, ADA

- Lansky score <60%

- Patient with expected survival <4 weeks (including disseminated CMV infection
involving lungs and/or CNS)

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Percent engraftment of donor B-cells in blood by STR testing

Outcome Description:

We will measure whether we are able to detect donor B-cells in the patient's blood after HSCT.

Outcome Time Frame:

1 Year

Safety Issue:


Principal Investigator

Christopher C Dvorak, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

University of California, San Francisco


United States: Institutional Review Board

Study ID:




Start Date:

August 2010

Completion Date:

September 2013

Related Keywords:

  • Severe Combined Immunodeficiency
  • SCID
  • Transplant
  • Alemtuzumab
  • Plerixafor
  • Immunologic Deficiency Syndromes
  • Severe Combined Immunodeficiency



UCSF Benioff Children's HospitalSan Francisco, California  94143