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

Transfusion Dependent Beta-Thalassaemia Clinical Trial

— ET-01  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04925206
• Other study ID #: EDG-001
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: August 17, 2021
• Est. completion date: June 30, 2024

Study information

• Verified date: February 2023
• Source: EdiGene (GuangZhou) Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 8
• Est. completion date: June 30, 2024
• Est. primary completion date: June 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 35 Years

Eligibility

Key Inclusion Criteria:

• Subjects and/or legal representative fully understand and voluntarily sign informed consent forms.

• Diagnosis of transfusion dependent ß-thalassemia (ß-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 a-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.

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia
• Transfusion Dependent Beta-Thalassaemia

Intervention

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

Locations

Country	Name	City	State
China	Guangzhou Women and Children's Medical Center	Guangzhou	Guangdong
China	Nanfang Hospital of Southern Medical University	Guangzhou	Guangdong
China	Shenzhen Children's Hospital	Shenzhen	Guangdong
China	Institute of Hematology & Blood Diseases Hospital	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
EdiGene (GuangZhou) Inc.

Country where clinical trial is conducted

China, 

Outcome

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