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
NCT: NCT06287086 ·
Status: NOT YET RECRUITING ·
Phase: N/A
· Sponsor: Bioray Laboratories
· Started: 2024-06-14
· Est. Completion: 2026-06-10
Official 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)
Study Design
- Study Type: INTERVENTIONAL
- Allocation: NA
- Model: SINGLE_GROUP
- Masking: NONE
- Enrollment: 1 participants
Interventions
- DRUG: BRL-101 autologous hematopoietic stem and progenitor cells injection — CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A gene.
Primary Outcomes
- Proportion of stem cell engrafted subjects (Within 42 Days After BRL-101 Infusion)
- Time to neutrophil engraftment (Within 42 Days After BRL-101 Infusion)
- Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion.. (Within 12 Months After BRL-101 Infusion)
More Sickle Cell Disease Trials
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.