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

Not Applicable

Active, not recruiting

• Conditions: Transfusion Dependent Beta-Thalassaemia
• Interventions: Biological: ET-01

• Registration Number: NCT04390971
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

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.

Study Timeline

• Study First Submitted: 2020-05-12
• Study First Submitted QC: 2020-05-12
• Study First Posted: 2020-05-18
• Study Start: 2023-02-10
• Status Verified: 2023-11
• Last Update Submitted: 2023-11-23
• Last Update Posted: 2023-11-27
• Primary Completion: 2025-06-15
• Study Completion: 2025-08-15

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 3

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ET-01	ET-01	BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.

Primary Outcome Measures

Name	Time	Method
Incidence of transplant-related mortality	Within 100 days post-transplant
Proportion of subjects with engraftment	Up to 42 days post-transplant
Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.	From ET-01 infusion to 104 weeks post-transplant
All-cause mortality	From signing of informed consent to 104 weeks post-transplant

Secondary Outcome Measures

Name	Time	Method
Change of total hemoglobin from baseline	Within 104 weeks post-transplant
Change of HbF from baseline	Within 104 weeks post-transplant
Change of proportion of HbF/Hb	Within 104 weeks post-transplant
Change of frequency of packed RBC transfusions	From 6 months before recruitment to 104 weeks post-transplant
Change of volume of packed RBC transfusions	From 6 months before recruitment to 104 weeks post-transplant

Trial Locations

Locations (3)

The Affiliated Hospital of Guizhou Medical University; 🇨🇳 Guiyang, Guizhou, China

Affiliated Hospital of Zunyi Medical University; 🇨🇳 Zunyi, Guizhou, China

Institute of Hematology & Blood Diseases Hospital; 🇨🇳 Tianjin, Tianjin, China