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

Transfusion Dependent Beta-Thalassaemia Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05752123
• Other study ID #: EDI-001-002
• Status: Active, not recruiting
• Phase: N/A
• Start date: February 18, 2023
• Est. completion date: August 15, 2025

Study information

• Verified date: February 2023
• Source: EdiGene (GuangZhou) Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 2
• Est. completion date: August 15, 2025
• Est. primary completion date: June 15, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 35 Years

Eligibility

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 ß-thalassemia (ß-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 a-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.

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia
• Transfusion Dependent Beta-Thalassaemia

Intervention

Biological:
• ET-01
Recruited participants will receive ET-01 IV infusion after conditioning.

Locations

Country	Name	City	State
China	PLA 923 Hospital	Nanning	Guangxi

Sponsors (2)

Lead Sponsor	Collaborator
EdiGene (GuangZhou) Inc.	PLA 923 Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.		From ET-01 infusion to 104 weeks post-transplant
Primary	All-cause mortality.		From signing of informed consent to 104 weeks post-transplant
Primary	Incidence of transplant-related mortality.		within 100 days post-transplant
Primary	Proportion of subjects with engraftment.		up to 42 days post-transplant
Secondary	Change in total hemoglobin from baseline.		within 104 weeks post-transplant
Secondary	Change of HbF from baseline.		within 104 weeks post-transplant
Secondary	Change of proportion of HbF/Hb.		within 104 weeks post-transplant
Secondary	Change of frequency of packed RBC transfusions.		From 6 months before recruitment to 104 weeks post-transplant
Secondary	Change of volume of packed RBC transfusions.		From 6 months before recruitment to 104 weeks post-transplant