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

Sponsor

EdiGene (GuangZhou) Inc. (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04925206

Collaborator

(none)

Enrollment

Locations

Arm

34.4

Anticipated Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Transfusion Dependent Beta-Thalassaemia	Biological: ET-01	Phase 1

Study Design

Study Type:

Interventional

Anticipated Enrollment :

8 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Aug 17, 2021

Anticipated Primary Completion Date :

Jun 30, 2024

Anticipated Study Completion Date :

Jun 30, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ET-01	Biological: ET-01 Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan.

Arm

Intervention/Treatment

Experimental: ET-01

Biological: ET-01

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

Outcome Measures

Primary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 35 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 exclusion criteria may apply.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Nanfang Hospital of Southern Medical University	Guangzhou	Guangdong	China	510515
2	Guangzhou Women and Children's Medical Center	Guangzhou	Guangdong	China	510623
3	Shenzhen Children's Hospital	Shenzhen	Guangdong	China	518035
4	Institute of Hematology & Blood Diseases Hospital	Tianjin	Tianjin	China	300020

Sponsors and Collaborators

EdiGene (GuangZhou) Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

EdiGene (GuangZhou) Inc.

ClinicalTrials.gov Identifier:

NCT04925206

Other Study ID Numbers:

EDG-001

First Posted:

Jun 14, 2021

Last Update Posted:

May 11, 2022

Last Verified:

May 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by EdiGene (GuangZhou) Inc.

Beta-Thalassaemia

CRISPR-Cas9

Human Hematopoietic Stem and Progenitor Cells

Additional relevant MeSH terms:

Thalassemia

beta-Thalassemia

Study Results

No Results Posted as of May 11, 2022