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

Primary Purpose

Transfusion Dependent Beta-Thalassaemia

Status

Recruiting

Phase

Not Applicable

Locations

China

Study Type

Interventional

Intervention

ET-01

About this trial

This is an interventional treatment trial for Transfusion Dependent Beta-Thalassaemia focused on measuring Beta-Thalassaemia, Hematopoietic Stem-Cell Transplantation

Eligibility Criteria

6 Years - 35 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
6~35 years old, all gender, weight ≥ 30kg;
Genetically diagnosed β-Thalassaemia;
Diagnosed as transfusion-dependent;
Eligible for autologous stem cell transplant;
Organs in good function;
Other protocol defined Inclusion criteria may apply.

Exclusion Criteria:

Thalassemia gene type is β0/β0;
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;
Other protocol defined Exclusion criteria may apply.

Sites / Locations

The Affiliated Hospital of Guizhou Medical UniversityRecruiting
Affiliated Hospital of Zunyi Medical UniversityRecruiting
Institute of Hematology & Blood Diseases HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ET-01

Arm Description

BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.

Outcomes

Primary Outcome Measures

Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.

All-cause mortality

Incidence of transplant-related mortality

Proportion of subjects with engraftment

Secondary Outcome Measures

Change of total hemoglobin from baseline

Change of HbF from baseline

Change of proportion of HbF/Hb

Change of frequency of packed RBC transfusions

Change of volume of packed RBC transfusions

Full Information

NCT ID

NCT04390971

First Posted

May 12, 2020

Last Updated

May 9, 2023

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Collaborators

EdiGene Inc., The Affiliated Hospital Of Guizhou Medical University, Zunyi Medical College

1. Study Identification

Unique Protocol Identification Number

NCT04390971

Brief Title

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

Official Title

A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia

Study Type

Interventional

2. Study Status

Record Verification Date

May 2023

Overall Recruitment Status

Recruiting

Study Start Date

February 10, 2023 (Actual)

Primary Completion Date

June 15, 2025 (Anticipated)

Study Completion Date

August 15, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Collaborators

EdiGene Inc., The Affiliated Hospital Of Guizhou Medical University, Zunyi Medical College

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Transfusion Dependent Beta-Thalassaemia

Keywords

Beta-Thalassaemia, Hematopoietic Stem-Cell Transplantation

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

6 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

ET-01

Arm Type

Experimental

Arm Description

BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.

Intervention Type

Biological

Intervention Name(s)

ET-01

Intervention Description

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

Primary Outcome Measure Information:

Title

Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.

Time Frame

From ET-01 infusion to 104 weeks post-transplant

Title

All-cause mortality

Time Frame

From signing of informed consent to 104 weeks post-transplant

Title

Incidence of transplant-related mortality

Time Frame

Within 100 days post-transplant

Title

Proportion of subjects with engraftment

Time Frame

Up to 42 days post-transplant

Secondary Outcome Measure Information:

Title

Change of total hemoglobin from baseline

Time Frame

Within 104 weeks post-transplant

Title

Change of HbF from baseline

Time Frame

Within 104 weeks post-transplant

Title

Change of proportion of HbF/Hb

Time Frame

Within 104 weeks post-transplant

Title

Change of frequency of packed RBC transfusions

Time Frame

From 6 months before recruitment to 104 weeks post-transplant

Title

Change of volume of packed RBC transfusions

Time Frame

From 6 months before recruitment to 104 weeks post-transplant

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

35 Years

Accepts Healthy Volunteers

Eligibility Criteria

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. Other protocol defined inclusion criteria may apply. 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. Other protocol defined exclusion criteria may apply.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Jun Shi, PhD

Phone

(86)2223900913

shijun@ihcams.ac.cn

First Name & Middle Initial & Last Name or Official Title & Degree

Jingyu Zhao, MPH

Phone

(86)13752253515

zhaojingyu@ihcams.ac.cn

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jun Shi, PhD

Organizational Affiliation

Institute of Hematology & Blood Diseases Hospital, China

Official's Role

Principal Investigator

Facility Information:

Facility Name

The Affiliated Hospital of Guizhou Medical University

City

Guiyang

State/Province

Guizhou

ZIP/Postal Code

550004

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jiao Jin, MD

Phone

(0851)86777812

jinjiao999@163.com

Facility Name

Affiliated Hospital of Zunyi Medical University

City

Zunyi

State/Province

Guizhou

ZIP/Postal Code

563000

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Yan Chen, MD

Phone

(0851)28609207

cyz600@163.com

Facility Name

Institute of Hematology & Blood Diseases Hospital

City

Tianjin

State/Province

Tianjin

ZIP/Postal Code

300020

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jun Shi

12. IPD Sharing Statement

Plan to Share IPD

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

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