A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.

Study Overview
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Brief Summary
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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.

Official Title
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A Single Site, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia

Conditions
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Transfusion Dependent Beta-Thalassaemia

Intervention / Treatment
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* Biological: ET-01


Participation Criteria
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Eligibility Criteria
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Key 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 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.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 35 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ET-01<br>BCL11A Enhancer modified Autologous Hematopoietic Stem Cells | Biological: ET-01<br>* Recruited participants will receive ET-01 IV infusion after conditioning.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Incidence of transplant-related mortality. |  | within 100 days post-transplant | 
| Proportion of subjects with engraftment. |  | up to 42 days post-transplant | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in 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 |