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

Primary Purpose

Beta-Thalassemia

Status

Recruiting

Phase

Phase 1

Locations

China

Study Type

Interventional

Intervention

BRL-101

About this trial

This is an interventional treatment trial for Beta-Thalassemia focused on measuring Transfusion-Dependent β-Thalassemia

Eligibility Criteria

3 Years - 35 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Nanfang Hospital, Southern Medical University
The First Affiliated Hospital of Guangxi Medical UniversityRecruiting
Xiangya Hospital of Central South University
Blood Diseases Hospital, Chinese Academy of Medical Sciences

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BRL-101

Arm Description

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.

Outcomes

Primary Outcome Measures

Proportion of subjects achieving successful neutrophil engraftment within 42 days after BRL-101 infusion

Time to neutrophil engraftment

Time to platelet engraftment

Incidence of Transplant-related Mortality (TRM) Within 100 Days and 12 months after BRL-101Infusion

Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion

Secondary Outcome Measures

Full Information

NCT ID

NCT05577312

First Posted

October 9, 2022

Last Updated

November 7, 2022

Sponsor

Bioray Laboratories

1. Study Identification

Unique Protocol Identification Number

NCT05577312

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Recruiting

Study Start Date

November 1, 2022 (Actual)

Primary Completion Date

August 4, 2025 (Anticipated)

Study Completion Date

September 10, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Bioray Laboratories

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Beta-Thalassemia

Keywords

Transfusion-Dependent β-Thalassemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

9 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

BRL-101

Arm Type

Experimental

Arm Description

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.

Intervention Type

Drug

Intervention Name(s)

BRL-101

Intervention Description

BRL-101 autologous hematopoietic stem and progenitor cells injection

Primary Outcome Measure Information:

Title

Proportion of subjects achieving successful neutrophil engraftment within 42 days after BRL-101 infusion

Time Frame

Within 42 Days After BRL-101 Infusion

Title

Time to neutrophil engraftment

Time Frame

Up to 12 Months After BRL-101 Infusion

Title

Time to platelet engraftment

Time Frame

Up to 12 Months After BRL-101 Infusion

Title

Incidence of Transplant-related Mortality (TRM) Within 100 Days and 12 months after BRL-101Infusion

Time Frame

Within 100 days and 12 months After BRL-101 Infusion

Title

Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion

Time Frame

Up to 12 Months After BRL-101 Infusion

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Maximum Age & Unit of Time

35 Years

Accepts Healthy Volunteers

Eligibility 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/Exclusion criteria may apply.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Wei Li

Phone

021-64340008

wli@brlmed.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Wei Li

Organizational Affiliation

Bioray Laboratories

Official's Role

Study Chair

Facility Information:

Facility Name

Nanfang Hospital, Southern Medical University

City

Guangzhou

State/Province

Guangdong

ZIP/Postal Code

510510

Country

China

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Xiaoqin Feng

First Name & Middle Initial & Last Name & Degree

Xiaoqin Feng

Facility Name

The First Affiliated Hospital of Guangxi Medical University

City

Nanning

State/Province

Guangxi

ZIP/Postal Code

530021

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Yongrong Lai

First Name & Middle Initial & Last Name & Degree

Yongrong Lai

Facility Name

Xiangya Hospital of Central South University

City

Changsha

State/Province

Hunan

ZIP/Postal Code

510510

Country

China

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Yajing Xu

First Name & Middle Initial & Last Name & Degree

Yajing Xu

Facility Name

Blood Diseases Hospital, Chinese Academy of Medical Sciences

City

Tianjin

State/Province

Tianjin

Country

China

Individual Site Status

Not yet recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Shi Jun

First Name & Middle Initial & Last Name & Degree

Shi Jun

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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