NCT02127892

SCID Bu/Flu/ATG Study With T Cell Depletion

Phase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling Donor

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Neena Kapoor, M.D. · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with a diagnosis of Severe Combined Immune Deficiency (SCID) who do not have an HLA-matched sibling donor. The stem cells will be derived from a 1) matched unrelated donor (MUD), 2) unrelated cord blood donor, or 3) a haplo-identical (parental) donor (in descending order of preference).Patients will receive a novel conditioning regimen with Busulfan, Fludarabine and Anti-thymocyte globulin (ATG) followed by an unrelated donor hematopoietic stem cell transplant (HSCT) with T-cell depletion using the CliniMACS device.

Detailed description

The study is being conducted to assess the following: * overall survival * event-free survival (events are defined as: death,non-engraftment/2nd transplant, immune reconstitution failure) * acute toxicity of the conditioning regimen * engraftment frequency immune reconstitution frequency and tempo acute and chronic graft-versus-host disease (GVHD), frequency and severity. The outcome from this protocol will be compared to the retrospective cohort consisting of all patients who have undergone haplo-identical HSCT for SCID at CHLA from 1984-2006 based on the assessment of the above-listed endpoints. The CliniMACS device will be used for CD34+ selection in place of the Isolex 300i. The CliniMACS CD34 Reagent System is an investigational medical device that has not yet been approved by the FDA. This device is used in vitro to select and enrich specific cell populations. When using the CliniMACS CD34 Reagent, the system selects CD34+ cells from heterogenous hematological cell populations for transplantation in cases where this is clinically indicated.

Conditions

• Severe Combined Immunodeficiency

Interventions

Type	Name	Description
BIOLOGICAL	unrelated BM with T cell depletion	Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
BIOLOGICAL	unrelated cord blood	Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
BIOLOGICAL	haplo BM with T cell depletion	haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
DEVICE	unrelated PBSC with T cell depletion	peripheral blood stem cell will be processed for CD34+ cell isolation.

Timeline

• Start date: 2007-01-02
• Primary completion: 2016-08-01
• Completion: 2016-08-01
• First posted: 2014-05-01
• Last updated: 2017-09-18
• Results posted: 2017-09-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated device study