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

A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia

Status

Terminated

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04925206

Acronym

ET-01

Enrollment

Registered

2021-06-14

Start date

2021-08-17

Completion date

2025-03-12

Last updated

2025-03-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transfusion Dependent Beta-Thalassaemia

Keywords

Beta-Thalassaemia, CRISPR-Cas9, Human Hematopoietic Stem and Progenitor Cells

Brief 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.

Interventions

BIOLOGICALET-01

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

12 Years to 35 Years

Healthy volunteers

Inclusion criteria

Key Inclusion Criteria: * Subjects and/or legal representative fully understand and voluntarily sign informed consent forms. * Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol. * Be not appropriate to receive conventional allogeneic hematopoietic stem cell transplantation. * Lansky/Karnofsky score ≥ 70%. * Eligible for hematopoietic stem cell transplantation and conditioning with busulfan as per investigator's judgement. Other protocol defined inclusion criteria may apply. Key

Exclusion criteria

* Subjects with associated α-thalassemia. * Subjects with any clinically significant acute or uncontrolled infections. * History of uncontrolled epilepsy or other mental disorders. * Previous treatment with allogeneic bone marrow transplantation or gene therapy. Other protocol defined

Design outcomes

Primary

Measure	Time frame
Frequency and severity of collected AEs & SAEs	Within 24 months after ET-01 infusion
All-cause mortality	From signing of informed consent up to 24 months post-ET-01 infusion
Incidence of transplant-related mortality	From baseline (pre-transfusion) up to 12 months post-ET-01 infusion
Total lymphocyte count	Within 24 months after ET-01 infusion
Proportion of subjects with abnormal proliferation of blood cells	Within 24 months after ET-01 infusion

Countries

China

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026