A Study to Evaluate the Safety and Efficacy 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: NCT05752123
• Lead Sponsor: EdiGene (GuangZhou) Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Status Verified: 2023-02
• Study First Submitted: 2023-02-16
• Study Start: 2023-02-18
• Study First Submitted QC: 2023-02-21
• Study First Posted: 2023-03-02
• Last Update Submitted: 2023-06-06
• Last Update Posted: 2023-06-08
• Primary Completion: 2025-06-15
• Study Completion: 2025-08-15

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 2

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;
• Organs in good function.

Other protocol defined Inclusion criteria may apply.

Key

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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.

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

Secondary Outcome Measures

Name	Time	Method
Change of proportion of HbF/Hb.	within 104 weeks post-transplant
Change of HbF from baseline.	within 104 weeks post-transplant
Change in total hemoglobin from baseline.	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 (1)

PLA 923 Hospital; 🇨🇳 Nanning, Guangxi, China