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Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
Sponsor: Bioray Laboratories
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)
Official title: A Phase 1/2 Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
Key Details
Gender
All
Age Range
3 Years - 35 Years
Study Type
INTERVENTIONAL
Enrollment
39
Start Date
2022-11-01
Completion Date
2027-07-10
Last Updated
2026-01-13
Healthy Volunteers
No
Conditions
Interventions
BRL-101
CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A enhancer site
Locations (4)
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, China
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
Xiangya Hospital of Central South University
Changsha, Hunan, China
Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China