drugset / Trial / NCT05577312
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
Phase 1/2
Enrolling by invitation
39 enrolled
Bioray Laboratories
Chinese Academy of Medical Sciences · collabFirst Affiliated Hospital of Guangxi Medical University · collabNanfang Hospital, Southern Medical University · collabXiangya Hospital of Central South University · collab
NaSingle-groupOpen-labelTreatment
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)
Timeline
- Start
- 2022-11-01
- Primary completion
- 2026-08-20
- Completion
- 2027-07-10
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | BRL-101 | Cell therapy | 3 cells/kg | Intravenous |