NCT04390971

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 6 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Transfusion Dependent Beta-Thalassaemia

Interventions

Type	Name	Description
BIOLOGICAL	ET-01	Recruited participants will receive ET-01 IV infusion after conditioning.

Timeline

• Start date: 2023-02-10
• Primary completion: 2023-03-05
• Completion: 2023-03-05
• First posted: 2020-05-18
• Last updated: 2026-02-11

Locations

3 sites across 1 country: China