A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia

Phase 1

Active, not recruiting

• Conditions: Transfusion Dependent Beta-Thalassaemia
• Interventions: Biological: ET-01

• Registration Number: NCT04925206
• Lead Sponsor: EdiGene (GuangZhou) Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-06-04
• Study First Submitted QC: 2021-06-11
• Study First Posted: 2021-06-14
• Study Start: 2021-08-17
• Status Verified: 2023-02
• Last Update Submitted: 2023-02-15
• Last Update Posted: 2023-02-17
• Primary Completion: 2024-06-30
• Study Completion: 2024-06-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 8

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

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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 exclusion criteria may apply.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ET-01	ET-01	-

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (4)

Nanfang Hospital of Southern Medical University; 🇨🇳 Guangzhou, Guangdong, China

Shenzhen Children's Hospital; 🇨🇳 Shenzhen, Guangdong, China

Institute of Hematology & Blood Diseases Hospital; 🇨🇳 Tianjin, Tianjin, China

Guangzhou Women and Children's Medical Center; 🇨🇳 Guangzhou, Guangdong, China