Clinical trial NCT04390971: Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Transfusion Dependent Beta-Thalassaemia
Keywords: Beta-Thalassaemia
Hematopoietic Stem-Cell Transplantation
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
Collaborators: name: EdiGene Inc.
name: The Affiliated Hospital Of Guizhou Medical University
name: Zunyi Medical College
First posted: 2020-05-18
Enrollment: 3
Sex: ALL
Minimum age (years): 6
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

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;
* 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.

Primary outcomes:
measure: Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.
timeFrame: From ET-01 infusion to 104 weeks post-transplant
measure: All-cause mortality
timeFrame: From signing of informed consent to 104 weeks post-transplant
measure: Incidence of transplant-related mortality
timeFrame: Within 100 days post-transplant
measure: Proportion of subjects with engraftment
timeFrame: Up to 42 days post-transplant
Secondary outcomes:
measure: Change of total hemoglobin from baseline
timeFrame: Within 104 weeks post-transplant
measure: Change of HbF from baseline
timeFrame: Within 104 weeks post-transplant
measure: Change of proportion of HbF/Hb
timeFrame: Within 104 weeks post-transplant
measure: Change of frequency of packed RBC transfusions
timeFrame: From 6 months before recruitment to 104 weeks post-transplant
measure: Change of volume of packed RBC transfusions
timeFrame: From 6 months before recruitment to 104 weeks post-transplant
Other outcomes:
Locations:
city: Guiyang
country: China
facility: The Affiliated Hospital of Guizhou Medical University
state: Guizhou
city: Zunyi
country: China
facility: Affiliated Hospital of Zunyi Medical University
state: Guizhou
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital
state: Tianjin
Study officials:
affiliation: Institute of Hematology & Blood Diseases Hospital, China
name: Jun Shi, PhD
role: PRINCIPAL_INVESTIGATOR
References: