Clinical Trial

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Study acronym: ExpMACs
Recruiting Early Phase 1
View on ClinicalTrials.gov →
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.
Protocol Amendment History 16 amendments
This ClinicalTrials.gov record has been amended 16 times since 2015-02-04; most recent amendment 2026-02-18.
Trial Details
NCT Number NCT02356653
Lead Sponsor Children's Hospital of Philadelphia
Conditions Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
Enrollment 100 participants
Start Date 2013-12
Primary Completion 2027-01 (estimated)
Study Completion 2030-01 (estimated)
Updated on ClinicalTrials.gov 2026-02-20