drugset / Trial / NCT06287086
Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease
Unknown
1 enrolled
Bioray Laboratories
Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College · collab
NaSingle-groupOpen-labelTreatment
Summary
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). 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
- 2024-06-14
- Primary completion
- 2025-08-20
- Completion
- 2026-06-10
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | BRL-101 | Cell therapy | — | Intravenous |