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

Transfusion Dependent Beta-Thalassaemia Clinical Trial

Official title:

A Multi-center, 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: NCT04390971
• Other study ID #: EDI-001
• Status: Active, not recruiting
• Phase: N/A
• Start date: February 10, 2023
• Est. completion date: August 15, 2025

Study information

• Verified date: November 2023
• Source: Institute of Hematology & Blood Diseases Hospital, China
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 3
• 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

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;

• Eligible for autologous stem cell transplant;

• Organs in good function.

• Other protocol defined inclusion criteria may apply.

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	The Affiliated Hospital of Guizhou Medical University	Guiyang	Guizhou
China	Institute of Hematology & Blood Diseases Hospital	Tianjin	Tianjin
China	Affiliated Hospital of Zunyi Medical University	Zunyi	Guizhou

Sponsors (4)

Lead Sponsor	Collaborator
Institute of Hematology & Blood Diseases Hospital, China	EdiGene Inc., The Affiliated Hospital Of Guizhou Medical University, Zunyi Medical College

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 of 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