A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.
Study Details
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
N/A |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: ET-01 BCL11A Enhancer modified Autologous Hematopoietic Stem Cells |
Biological: ET-01
Recruited participants will receive ET-01 IV infusion after conditioning.
|
Outcome Measures
Primary Outcome Measures
- 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
- 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]
Eligibility Criteria
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | PLA 923 Hospital | Nanning | Guangxi | China | 530021 |
Sponsors and Collaborators
- EdiGene (GuangZhou) Inc.
- PLA 923 Hospital
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- EDI-001-002