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

A Phase 1/2 Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05577312

Enrollment

Registered

2022-10-13

Start date

2022-11-02

Completion date

2027-07-10

Last updated

2026-07-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Beta-Thalassemia

Keywords

Transfusion-Dependent β-Thalassemia

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)

Detailed description

This clinical trial is a multi-center, single-arm, single-dose, open-label study without dose escalation. The proposed dose is ≥ 3 × 106 CD34 + cells/kg administered as a single intravenous infusion. The primary objective of Phase 1 is to explore the safety of the study drug in different age groups. For subjects of each age group, myeloablative conditioning and dosing of the remaining subjects was initiated only after completion of dosing and safety observations and assessments in sentinel subjects. The Phase 2 primary objective was to determine the effectiveness of BRL-101 administered intravenously to patients with TDT.

Interventions

DRUGBRL-101

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

3 Years to 35 Years

Healthy volunteers

Inclusion 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/

Design outcomes

Primary

Measure	Time frame	Description
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

Countries

China

Contacts

STUDY_CHAIRRongmei Liang, PhD

Bioray Laboratories

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 22, 2026