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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE1/PHASE2

Total Enrollment

39 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-01

Study Completion Date

2027-07-10

Brief Summary

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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)

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Detailed Description

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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.

Conditions

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Beta-Thalassemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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BRL-101

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.

Group Type EXPERIMENTAL

BRL-101

Intervention Type DRUG

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

Interventions

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BRL-101

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

Intervention Type DRUG

Other Intervention Names

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BRL-101 autologous hematopoietic stem and progenitor cells injection

Eligibility Criteria

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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.

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).

Minimum Eligible Age

3 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No