This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Proportion of stem cell engrafted subjects
Timeframe: Within 42 Days After BRL-101 Infusion
Time to neutrophil engraftment
Timeframe: Up to 12 Months After BRL-101 Infusion
Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion
Timeframe: Up to 12 Months After BRL-101 Infusion