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

Sponsor

Bioray Laboratories (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05577312

Collaborator

(none)

Enrollment

Locations

Arm

46.3

Anticipated Duration (Months)

2.3

Patients Per Site

Patients Per Site Per Month

Study Details

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)

Condition or Disease	Intervention/Treatment	Phase
Beta-Thalassemia	Drug: BRL-101	Phase 1

Study Design

Study Type:

Interventional

Anticipated Enrollment :

9 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Nov 1, 2022

Anticipated Primary Completion Date :

Aug 4, 2025

Anticipated Study Completion Date :

Sep 10, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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.	Drug: BRL-101 BRL-101 autologous hematopoietic stem and progenitor cells injection

Arm

Intervention/Treatment

Experimental: 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.

Drug: BRL-101

BRL-101 autologous hematopoietic stem and progenitor cells injection

Outcome Measures

Primary Outcome Measures

Proportion of subjects achieving successful neutrophil engraftment within 42 days after BRL-101 infusion [Within 42 Days After BRL-101 Infusion]
Time to neutrophil engraftment [Up to 12 Months After BRL-101 Infusion]
Time to platelet engraftment [Up to 12 Months After BRL-101 Infusion]
Incidence of Transplant-related Mortality (TRM) Within 100 Days and 12 months after BRL-101Infusion [Within 100 days and 12 months After BRL-101 Infusion]
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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 35 Years

Sexes Eligible for Study:

All

Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Nanfang Hospital, Southern Medical University	Guangzhou	Guangdong	China	510510
2	The First Affiliated Hospital of Guangxi Medical University	Nanning	Guangxi	China	530021
3	Xiangya Hospital of Central South University	Changsha	Hunan	China	510510
4	Blood Diseases Hospital, Chinese Academy of Medical Sciences	Tianjin	Tianjin	China

Sponsors and Collaborators

Bioray Laboratories

Investigators

Study Chair: Wei Li, Bioray Laboratories

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Bioray Laboratories

ClinicalTrials.gov Identifier:

NCT05577312

Other Study ID Numbers:

2022-BRL-101

First Posted:

Oct 13, 2022

Last Update Posted:

Nov 10, 2022

Last Verified:

Nov 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Bioray Laboratories

Transfusion-Dependent β-Thalassemia

Additional relevant MeSH terms:

Thalassemia

beta-Thalassemia

Study Results

No Results Posted as of Nov 10, 2022