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Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major

NCT06065189 · Status: COMPLETED · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2026-05-15

No results posted yet for this study

Summary

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.

Conditions

Interventions

BIOLOGICAL

CS-101 injection

Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

Sponsors & Collaborators

  • CorrectSequence Therapeutics Co., Ltd

    collaborator INDUSTRY

  • Children's Hospital of Fudan University

    lead OTHER

Principal Investigators

  • Xiaowen Zhai, M.D. · Children's Hospital of Fudan University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-11-07
Primary Completion
2025-08-12
Completion
2025-08-12

Countries

  • China

Study Locations

More Related Trials

Entities

Diseases

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 NCT06065189 on ClinicalTrials.gov