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

NCT05577312 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 39

Last updated 2026-01-13

No results posted yet for this study

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)

Conditions

Interventions

DRUG

BRL-101

CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A enhancer site

Sponsors & Collaborators

  • First Affiliated Hospital of Guangxi Medical University

    collaborator OTHER

  • Xiangya Hospital of Central South University

    collaborator OTHER

  • Chinese Academy of Medical Sciences

    collaborator OTHER

  • Nanfang Hospital, Southern Medical University

    collaborator OTHER

  • Bioray Laboratories

    lead INDUSTRY

Principal Investigators

  • Xiaochen Wang, PhD · Bioray Laboratories

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-11-01
Primary Completion
2026-08-20
Completion
2027-07-10

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05577312 on ClinicalTrials.gov