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

NCT05577312 | Enrolling By Invitation Phase 1

• 1 other identifier: 2022-BRL-101
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 4

Brief 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

Beta-Thalassemia

Keywords

Transfusion-Dependent β-Thalassemia

Outcome Measures

Primary Outcomes (3)

• Proportion of stem cell engrafted subjects

  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.

  Within 42 Days After BRL-101 Infusion

• Time to neutrophil engraftment

  Defined as Day 1 of absolute peripheral blood neutrophil count ≥ 0.5 × 109/L for 3 consecutive days

  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

  Adverse events assessed according to NCI-CTCAE v5.0 criteria

  Up to 12 Months After BRL-101 Infusion

Study Arms (1)

BRL-101

EXPERIMENTAL

BRL-101 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Subjects will receive a single infusion of BRL-101.

Drug: BRL-101

Interventions

BRL-101 DRUG

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

Also known as: BRL-101 autologous hematopoietic stem and progenitor cells injection

BRL-101

Eligibility Criteria

• Age: 3 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Bioray Laboratories: lead
• First Affiliated Hospital of Guangxi Medical University: collaborator
• Chinese Academy of Medical Sciences: collaborator
• Xiangya Hospital of Central South University: collaborator
• Nanfang Hospital, Southern Medical University: collaborator

Study Sites (4)

Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, 510510, China

Location

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, 530021, China

Location

Xiangya Hospital of Central South University

Changsha, Hunan, 510510, China

Location

Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Xiaochen Wang, PhD

  Bioray Laboratories

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 9, 2022
• First Posted: October 13, 2022
• Study Start: November 1, 2022
• Primary Completion (Estimated): August 20, 2026
• Study Completion (Estimated): July 10, 2027
• Last Updated: January 13, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (4)