A Safety and Efficacy Study Evaluating ET-01 in Subjects... | NCT04925206

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

NCT04925206

Sponsor

EdiGene (GuangZhou) Inc.

Status

Terminated

Last Update Posted

Mar 30, 2025Actual

Enrollment

8Actual

Phase

Phase 1

Conditions

Transfusion Dependent Beta-Thalassaemia

Interventions

ET-01

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2025

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Development plan change

Expanded Access Info

Start Date

Aug 17, 2021Actual

Primary Completion Date

Jan 30, 2024Actual

Completion Date

Mar 12, 2025Actual

First Submitted Date

Jun 4, 2021

First Submission Date that Met QC Criteria

Jun 11, 2021

First Posted Date

Jun 14, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 25, 2025

Last Update Posted Date

Mar 30, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

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 provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

8Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
ET-01	Experimental		Biological: ET-01

Interventions

Name	Type	Description	Arm Group Labels	Other Names
ET-01	Biological	Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan.	ET-01

Outcomes Module

Primary Outcomes

Measure	Description	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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility 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 exclusion criteria may apply.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

12 Years

Maximum Age

35 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Nanfang Hospital of Southern Medical University		Guangzhou	Guangdong	510515	China
Guangzhou Women and Children's Medical Center

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D017086	beta-Thalassemia

Ancestor Terms

ID	Term
D013789	Thalassemia
D000745	Anemia, Hemolytic, Congenital
D000743	Anemia, Hemolytic
D000740	Anemia

Browse Leaves

Not provided

Browse Branches

Not provided