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A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

NCT06291961 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2026-02-10

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 CS-101 in treating patients with β-thalassemia major anemia.

Conditions

  • Beta-Thalassemia Major

Interventions

GENETIC

CS-101 injection

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

Sponsors & Collaborators

  • CorrectSequence Therapeutics Co., Ltd

    lead INDUSTRY

Principal Investigators

  • Yaliang Li · CorrectSequence Therapeutics Co., Ltd

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-18
Primary Completion
2025-11-17
Completion
2025-11-17

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 NCT06291961 on ClinicalTrials.gov