NCT04925206: ET-01

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

Terminated Phase 1 Last updated 30 March 2025

What this trial tests

Phase 1 trial testing ET-01 in Transfusion Dependent Beta-Thalassaemia in 8 participants. Terminated before completion.

Timeline

17 August 2021

Primary endpoint
30 January 2024

12 March 2025

Quick facts

Lead sponsor	EdiGene (GuangZhou) Inc.
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	8
Start date	17 August 2021
Primary completion	30 January 2024
Estimated completion	12 March 2025
Sites	4 locations across China

Drugs / interventions tested

• ET-01 — full drug profile →

Conditions studied

• Transfusion Dependent Beta-Thalassaemia — all drugs for Transfusion Dependent Beta-Thalassaemia →

Sponsor

EdiGene (GuangZhou) Inc. — full company profile →

Who can join

Adults 12 to 35, any sex, with Transfusion Dependent Beta-Thalassaemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. CRISPR/Cas9 Delivery System Engineering for Genome Editing in Therapeutic Applications.
   Cheng H, Zhang F, Ding Y. · · 2021 · cited 59× · PMID 34683943 · DOI 10.3390/pharmaceutics13101649
2. CRISPR/Cas systems: Delivery and application in gene therapy.
   Huang J, Zhou Y, Li J, Lu A, et al · · 2022 · cited 47× · PMID 36483767 · DOI 10.3389/fbioe.2022.942325
3. CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments.
   Laurent M, Geoffroy M, Pavani G, Guiraud S. · · 2024 · cited 45× · PMID 38786024 · DOI 10.3390/cells13100800
4. Current trends of clinical trials involving CRISPR/Cas systems.
   Zhang S, Wang Y, Mao D, Wang Y, et al · · 2023 · cited 35× · PMID 38020120 · DOI 10.3389/fmed.2023.1292452
5. An Update on the Application of CRISPR Technology in Clinical Practice.
   Morshedzadeh F, Ghanei M, Lotfi M, Ghasemi M, et al · · 2024 · cited 33× · PMID 37269466 · DOI 10.1007/s12033-023-00724-z
6. The power and the promise of CRISPR/Cas9 genome editing for clinical application with gene therapy.
   Guo N, Liu JB, Li W, Ma YS, et al · · 2022 · cited 32× · PMID 36100322 · DOI 10.1016/j.jare.2021.11.018
7. Clinical progress in genome-editing technology and in vivo delivery techniques.
   Khirallah J, Eimbinder M, Li Y, Xu Q. · · 2023 · cited 26× · PMID 36669950 · DOI 10.1016/j.tig.2022.12.001
8. Current advances of CRISPR-Cas technology in cell therapy.
   Qiu HY, Ji RJ, Zhang Y. · · 2022 · cited 25× · PMID 37193354 · DOI 10.1016/j.cellin.2022.100067