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Clinical Trials in China / NCT05577312
Active, not recruiting Phase 1/2

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

NCT05577312 · tracked via the Priya Life Science China tracker
Sponsor
Bioray Laboratories
Phase
Phase 1/2
Started
2022-11-02
Last updated
2026-07-21

Condition(s) studied

Beta-Thalassemia

Investigational drug(s) / intervention(s)

BRL-101

BRL-101: CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A enhancer site

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)

Eligibility

Sex
ALL
Min age
3 Years
Max age
35 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Proportion of stem cell engrafted subjects — Within 42 Days After BRL-101 Infusion
    Stem cell engraftment was defined as an absolute peripheral blood neutrophil count of ≥ 0.5 × 109/L for 3 consecutive days within 42 days following BRL-101 intravenous infusion.
  • Time to neutrophil engraftment — Up to 12 Months After BRL-101 Infusion
    Defined as Day 1 of absolute peripheral blood neutrophil count ≥ 0.5 × 109/L for 3 consecutive days
  • Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion — Up to 12 Months After BRL-101 Infusion
    Adverse events assessed according to NCI-CTCAE v5.0 criteria

Trial sites (7)

FacilityCityRegionStatus
Nanfang Hospital, Southern Medical University Guangzhou Guangdong
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen Universit Guanzhou Guangdong
Shenzhen Children 's Hospital Shenzhen Guangdong
No. 923 Hospital of Joint Support Force of Chinese People 's Liberation Army Hospital Nanning Guangxi
The First Affiliated Hospital of Guangxi Medical University Nanning Guangxi
Xiangya Hospital of Central South University Changsha Hunan
Chinese Academy of Medical Sciences Tianjin Tianjin Municipality

More Bioray Laboratories trials in China

Universal CAR-T Cells (BRL-301) in Relapse or Refractory Autoimmune Diseases Enrolling by invitation
Universal CAR-T Cell Therapy for MM Recruiting
Long-term Follow-up Study of BRL-101 for TDT Recruiting
Long-term Follow-up Study of BHC001 for TDT Recruiting
The Safety and Efficacy of BRL-201 in the Treatment of r/r B Lymphocyte Non-Hodgkin Lymphoma Recruiting

Other trials for the same condition

A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia Recruiting
A Long-term Follow-up Study in Participants Who Received CS-101 Enrolling by invitation
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector Starting soon
A Long-term Follow-up Study in Participants Who Received CS-101 Recruiting
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia. Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05577312 on ClinicalTrials.gov ↗ ← All trials in China