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β-globin Restored Autologous HSC in β-thalassemia Major Patients

NCT04205435 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2022-10-13

No results posted yet for this study

Summary

This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.

Conditions

  • β-thalassemia Major

Interventions

BIOLOGICAL

β-globin restored autologous HSC

gene edited autologous hematopoietic stem cells with β-globin restoration

Sponsors & Collaborators

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

    collaborator OTHER

  • Bioray Laboratories

    lead INDUSTRY

Principal Investigators

  • Xinhua Zhang, Prof · The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army

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
2021-11-01
Primary Completion
2022-06-01
Completion
2022-07-25

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 NCT04205435 on ClinicalTrials.gov