Transfusion Dependent Beta-Thalassaemia
Conditions
Keywords
Beta-Thalassaemia, Hematopoietic Stem-Cell Transplantation
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.
Interventions
BIOLOGICALET-01
Recruited participants will receive ET-01 IV infusion after conditioning.
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
EdiGene Inc.
The Affiliated Hospital Of Guizhou Medical University
Zunyi Medical College
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 35 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
| Change of total hemoglobin from baseline | Within 104 weeks post-transplant |
| Change of HbF from baseline | Within 104 weeks post-transplant |
Countries
China
Contacts
PRINCIPAL_INVESTIGATORJun Shi, PhD
Institute of Hematology & Blood Diseases Hospital, China
Outcome results
None listed