Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells

NCT ID: NCT05442346

Last Updated: 2024-02-28

Basic Information

• Recruitment Status: SUSPENDED
• Clinical Phase: NA
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-12-25
• Study Completion Date: 2024-11-30

Brief Summary

This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.

Detailed Description

γ-globin reactivated autologous hematopoietic stem cells will be manufactured using Glycosylase Base Editors. Subject participation for this study will be 2 year. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 15 years post-transplant.

Conditions

Thalassemia Major

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

γ-globin reactivated autologous hematopoietic stem cells

each subject will accept one dose of γ-globin reactivated autologous hematopoietic stem cells

Group Type: EXPERIMENTAL

γ-globin reactivated autologous hematopoietic stem cells

Intervention Type: BIOLOGICAL

gene edited autologous hematopoietic stem cells with γ-globin expression; BRL-103

Interventions

γ-globin reactivated autologous hematopoietic stem cells

gene edited autologous hematopoietic stem cells with γ-globin expression; BRL-103

Intervention Type: BIOLOGICAL

Eligibility Criteria

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 EBV, HIV, CMV, TP, HAV, HBV and HCV.
• 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.
• Recently diagnosed as malaria.
• History of complex autoimmune disease.
• Persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value >3 X the upper limit of normal (ULN).
• Subjects with severe heart, lung and kidney diseases.
• With serious iron overload, serum ferritin>5000mg/ml.
• Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or Investigator.
• Subjects who are receiving treatment from another clinical study, or have received another gene therapy.
• Subjects or guardians had resisted the guidance of the attending doctor.
• Subjects whom the investigators do not consider appropriate for participating in this clinical study

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 35 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

First Affiliated Hospital of Guangxi Medical University

OTHER

Sponsor Role: collaborator

Bioray Laboratories

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

lai yongrong, PhD

Role: PRINCIPAL_INVESTIGATOR

First Affiliated Hospital of Guangxi Medical University

Locations

Shanghai Bioray Laboratories Inc

Shanghai, Shanghai Municipality, China

Countries

China

Other Identifiers

2021-BRL-103

Identifier Type: -

Identifier Source: org_study_id