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

NCT04211480 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-01-05

No results posted yet for this study

Summary

This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.

Conditions

  • β Thalassemia Major

Interventions

BIOLOGICAL

γ-globin reactivated autologous hematopoietic stem cells

gene edited autologous hematopoietic stem cells with γ-globin expression

Sponsors & Collaborators

  • Xiangya Hospital of Central South University

    collaborator OTHER

  • The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

    collaborator OTHER

  • Bioray Laboratories

    lead INDUSTRY

Principal Investigators

  • Bin Fu, Prof. · Xiangya Hospital Central University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-10-01
Primary Completion
2023-10-15
Completion
2023-11-27

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04211480 on ClinicalTrials.gov