NCT02356653

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sponsor: Children's Hospital of Philadelphia · Academic / Other
• Sex: All
• Age: 30 Years
• Healthy volunteers: Not accepted

Summary

The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia IRB approved protocols that utilize CliniMACs technology for T depletion.

Detailed description

Only 25-30% of patients who may benefit from HSCT have a matched related donor. There is a higher rate of complications using cells from an unrelated or partially matched related donor. T cells within the donor cells may cause a complication called graft vs. host disease (GVHD). The goal of this study is to use the CliniMACs device to remove the T cells that cause GVHD, called T cell depletion.

Conditions

• Leukemia
• Inborn Errors of Metabolism
• Bone Marrow Failure Syndromes
• Immunodeficiencies
• Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)	Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.

Timeline

• Start date: 2013-12-01
• Primary completion: 2027-01-01
• Completion: 2030-01-01
• First posted: 2015-02-05
• Last updated: 2026-02-20

Locations

1 site across 1 country: United States