Clinical Trial

Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia

Active, Not Recruiting Phase 1/2
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Summary
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)
Protocol Amendment History 3 changes
critical Trial status changed: Enrolling by Invitation → Active, Not Recruiting 2026-07-22
notable Trial sites expanded: 4 -> 7 locations 2026-07-22
notable Primary completion pushed: 2026-08-20 -> 2026-10-20 2026-07-22
Trial Details
NCT Number NCT05577312
Lead Sponsor Bioray Laboratories
Collaborators: First Affiliated Hospital of Guangxi Medical University, Xiangya Hospital of Central South University, Chinese Academy of Medical Sciences, Nanfang Hospital, Southern Medical University, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army, Shenzhen Children's Hospital
Conditions Beta-Thalassemia
Enrollment 39 participants
Start Date 2022-11-02
Primary Completion 2026-10-20 (estimated)
Study Completion 2027-07-10 (estimated)
Updated on ClinicalTrials.gov 2026-07-21