NCT06287086

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

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

• Status: Not Yet Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 1 (estimated)
• Sponsor: Bioray Laboratories · Industry
• Sex: All
• Age: 3 Years – 35 Years
• Healthy volunteers: Not accepted

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)

Detailed 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.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	BRL-101 autologous hematopoietic stem and progenitor cells injection	CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A gene.

Timeline

• Start date: 2024-06-14
• Primary completion: 2025-08-20
• Completion: 2026-06-10
• First posted: 2024-02-29
• Last updated: 2024-06-03