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

Institute of Hematology & Blood Diseases Hospital, China3 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Transfusion Dependent Beta-Thalassaemia

Interventions

ET-01BIOLOGICAL

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

Eligibility

Sex: ALLMin age: 6 YearsMax age: 35 Years

Medical Language ↔ Plain English

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 exclusion criteria may apply.

Locations (3)

The Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, China

Affiliated Hospital of Zunyi Medical University

Zunyi, Guizhou, China

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China

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

All-cause mortality

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

Incidence of transplant-related mortality

Time frame: Within 100 days post-transplant

Proportion of subjects with engraftment

Time frame: Up to 42 days post-transplant

Secondary Outcomes

Change of total hemoglobin from baseline

Time frame: Within 104 weeks post-transplant

Change of HbF from baseline

Time frame: Within 104 weeks post-transplant

Change of proportion of HbF/Hb

Time frame: Within 104 weeks post-transplant

Change of frequency of packed RBC transfusions

Time frame: From 6 months before recruitment to 104 weeks post-transplant

Change of volume of packed RBC transfusions

Time frame: From 6 months before recruitment to 104 weeks post-transplant

Data from ClinicalTrials.gov

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