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Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

NCT ID: NCT04390971

Last Updated: 2023-11-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

NA

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-10

Study Completion Date

2025-08-15

Brief Summary

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This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

Detailed Description

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After the proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. Two years follow-up will be carried out post-transplantation and related data will be collected. Subjects who have been treated by ET-01 will be asked to participate in a long-term follow up study. Monitoring the long-term efficacy and safety up to 15 years after ET-01 transplant.

Conditions

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Transfusion Dependent Beta-Thalassaemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ET-01

BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.

Group Type EXPERIMENTAL

ET-01

Intervention Type BIOLOGICAL

Recruited participants will receive ET-01 IV infusion after conditioning.

Interventions

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ET-01

Recruited participants will receive ET-01 IV infusion after conditioning.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
* 6\~35 years old, all gender;
* Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
* Eligible for autologous stem cell transplant;
* Eligible for autologous stem cell transplant;
* Organs in good function.

Exclusion Criteria

* Subjects with associated α-thalassemia;
* Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
* HLA identical sibling or unrelated donors are available;
* Prior allo-HSCT or gene therapy.
Minimum Eligible Age

6 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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EdiGene Inc.

INDUSTRY

Sponsor Role collaborator

The Affiliated Hospital Of Guizhou Medical University

OTHER

Sponsor Role collaborator

Zunyi Medical College

OTHER

Sponsor Role collaborator

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role lead

Responsible Party

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Jun Shi

Director of Regenerative Medicine Clinic Center

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Jun Shi, PhD

Role: PRINCIPAL_INVESTIGATOR

Institute of Hematology & Blood Diseases Hospital, China

Locations

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The Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, China

Site Status

Affiliated Hospital of Zunyi Medical University

Zunyi, Guizhou, China

Site Status

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China

Site Status

Countries

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China

Other Identifiers

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EDI-001

Identifier Type: -

Identifier Source: org_study_id