Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia

• Status: Not yet recruiting
• Trial end date: 2026-09-10
• Target enrollment: 0
• Participant gender: All

Summary

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)

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bioray Laboratories

Criteria

Key Inclusion Criteria:

• Fully understand and voluntarily sign informed consent. 3-35years old. At least one legal guardian and/or Subjects to sign informed consent.

• Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, β+β+, β+β0, βEβ0 genotype.

• Subjects with no affection with HIV, TP, HBV, HCV, CMV and EBV.

• Subjects body condition eligible for autologous stem cell transplant.

Key Exclusion Criteria:

• Subjects acceptable for allogeneic hematopoietic stem cell transplantation and have an available fully matched related donor.

• Active bacterial, viral, or fungal infection.

• Treated with erythropoietin prior 3 months.

• Immediate family member with any known hematological tumor.

• Subjects with severe psychiatric disorders to be unable to cooperate.

• Prior hematopoietic stem cell transplant (HSCT).

Other protocol defined Inclusion/Exclusion criteria may apply.