Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC

• Conditions: Leukemia; Bone Marrow Failure Syndrome; Immunodeficiencies

• Registration Number: NCT03145545
• Lead Sponsor: Children's Hospital of Philadelphia

Brief Summary

The primary objective 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 Institutional Review Board (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. An unrelated cord blood may not be available due to size or matching criteria, or if a reduced intensity regiment is recommended. The risk of severe graft vs. host disease (GVHD) and other complications is higher with unrelated donors, or partially matched related donors. At the Children's Hospital of Philadelphia (CHOP) there is extensive experience using mismatched unrelated donors or partially matched related donors with complete or partial T depletion to reduce the risk of severe GVHD.

Study Timeline

• Study First Submitted: 2017-05-05
• Study First Submitted QC: 2017-05-05
• Study First Posted: 2017-05-09
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-18
• Last Update Posted: 2025-05-22

Recruitment & Eligibility

• Status: AVAILABLE
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified

Trial Locations

Locations (1)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States