A Single Site, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassemia

EdiGene (GuangZhou) Inc.1 site in 1 country2 target enrollmentFebruary 18, 2023

ConditionsTransfusion Dependent Beta-Thalassaemia

InterventionsET-01

DrugsET-01

Overview

Phase: Not Applicable
Intervention: ET-01
Conditions: Transfusion Dependent Beta-Thalassaemia
Sponsor: EdiGene (GuangZhou) Inc.
Enrollment: 2
Locations: 1
Primary Endpoint: Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.
Status: Terminated
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

February 18, 2023

End Date

March 12, 2025

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

EdiGene (GuangZhou) Inc.

Responsible Party

Sponsor

Eligibility Criteria

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.

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.

Arms & Interventions

ET-01

BCL11A Enhancer modified Autologous Hematopoietic Stem Cells

Intervention: ET-01

Outcomes

Primary Outcomes

Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.

Time Frame: From ET-01 infusion to 104 weeks post-transplant

Incidence of transplant-related mortality.

Time Frame: within 100 days post-transplant

All-cause mortality.

Time Frame: From signing of informed consent to 104 weeks post-transplant

Proportion of subjects with engraftment.

Time Frame: up to 42 days post-transplant

Secondary Outcomes

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)