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Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:
Clinical Study on the Safety and Efficacy of a Single Intravenous Dose of CRISPR/Cas9-Edited Autologous CD34+ Hematopoietic Stem/Progenitor Cells (BRL-101) in the Treatment of Sickle Cell Disease

Clinical Trial 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)

Clinical Trial Description

This clinical trial is a single-arm, single-dose, single center, open-label study without dose escalation. The primary objective is to explore the safety of the study drug in SCD. Myeloablative conditioning and administration for the remaining subjects can only be started after the first subject completes dosing and safety observation and assessment. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06287086
Study type Interventional
Source Bioray Laboratories
Contact Jun shi, PhD
Phone 13752253515
Email shijun@ihcams.ac.cn
Status Not yet recruiting
Phase N/A
Start date June 14, 2024
Completion date June 10, 2026
View Details
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