Back to resultsA Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.
Primary Purpose
Transfusion Dependent Beta-Thalassaemia
Status
Active
Phase
Not Applicable
Locations
China
Study Type
Interventional
Intervention
ET-01
Sponsored by
About this trial
This is an interventional treatment trial for Transfusion Dependent Beta-Thalassaemia
Eligibility Criteria
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.
Sites / Locations
- PLA 923 Hospital
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 in 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
NCT05752123
First Posted
February 16, 2023
Last Updated
June 6, 2023
Sponsor
EdiGene (GuangZhou) Inc.
Collaborators
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
1. Study Identification
Unique Protocol Identification Number
NCT05752123
Brief Title
A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.
Official Title
A Single Site, 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
February 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
February 18, 2023 (Actual)
Primary Completion Date
June 15, 2025 (Anticipated)
Study Completion Date
August 15, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
EdiGene (GuangZhou) Inc.
Collaborators
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transfusion Dependent Beta-Thalassaemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)
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 in 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
No
Eligibility Criteria
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.
Facility Information:
Facility Name
PLA 923 Hospital
City
Nanning
State/Province
Guangxi
ZIP/Postal Code
530021
Country
China
12. IPD Sharing Statement
Learn more about this trial
A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.
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