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A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia

NCT04925206 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2025-03-30

No results posted yet for this study

Summary

This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.

Conditions

  • Transfusion Dependent Beta-Thalassaemia

Interventions

BIOLOGICAL

ET-01

Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan.

Sponsors & Collaborators

  • EdiGene (GuangZhou) Inc.

    lead INDUSTRY

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
2021-08-17
Primary Completion
2024-01-30
Completion
2025-03-12

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 NCT04925206 on ClinicalTrials.gov