NCT05752123

Brief 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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Feb 2023

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 16, 2023

Completed
2 days until next milestone

Study Start

First participant enrolled

February 18, 2023

Completed
12 days until next milestone

First Posted

Study publicly available on registry

March 2, 2023

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 29, 2024

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 12, 2025

Completed
Last Updated

March 30, 2025

Status Verified

March 1, 2025

Enrollment Period

12 months

First QC Date

February 16, 2023

Last Update Submit

March 25, 2025

Conditions

Transfusion Dependent Beta-Thalassaemia

Outcome Measures

Primary Outcomes (4)

  • Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.

    From ET-01 infusion to 104 weeks post-transplant

  • All-cause mortality.

    From signing of informed consent to 104 weeks post-transplant

  • Incidence of transplant-related mortality.

    within 100 days post-transplant

  • Proportion of subjects with engraftment.

    up to 42 days post-transplant

Secondary Outcomes (5)

  • Change in total hemoglobin from baseline.

    within 104 weeks post-transplant

  • Change of HbF from baseline.

    within 104 weeks post-transplant

  • Change of proportion of HbF/Hb.

    within 104 weeks post-transplant

  • Change of frequency of packed RBC transfusions.

    From 6 months before recruitment to 104 weeks post-transplant

  • Change of volume of packed RBC transfusions.

    From 6 months before recruitment to 104 weeks post-transplant

Study Arms (1)

ET-01

EXPERIMENTAL

BCL11A Enhancer modified Autologous Hematopoietic Stem Cells

Biological: ET-01

Interventions

ET-01BIOLOGICAL

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

ET-01

Eligibility Criteria

Age6 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
  • \~35 years old, all gender;
  • Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
  • Eligible for autologous stem cell transplant;
  • Organs in good function.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

PLA 923 Hospital

Nanning, Guangxi, 530021, China

Location

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 16, 2023

First Posted

March 2, 2023

Study Start

February 18, 2023

Primary Completion

January 29, 2024

Study Completion

March 12, 2025

Last Updated

March 30, 2025

Record last verified: 2025-03

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)