NCT06300723

Clinical Study of BRL-101 in Severe SCD

Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of CRISPR/Cas9-edited Autologous CD34 + Hematopoietic Stem/Progenitor Cells (BRL-101) in the Treatment of Severe Sickle Cell Disease

• Status: Enrolling By Invitation
• Phase: N/A
• Study type: Interventional
• Enrollment: 3 (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	CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A gene.

Timeline

• Start date: 2024-07-29
• Primary completion: 2026-03-20
• Completion: 2026-06-15
• First posted: 2024-03-08
• Last updated: 2024-07-31

Locations

1 site across 1 country: China