A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.

A Single Site, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassemia

Status

Terminated

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05752123

Enrollment

Registered

2023-03-02

Start date

2023-02-18

Completion date

2025-03-12

Last updated

2025-03-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transfusion Dependent Beta-Thalassaemia

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.

Interventions

BIOLOGICALET-01

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Years to 35 Years

Healthy volunteers

Inclusion criteria

Key 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; * Organs in good function. Other protocol defined Inclusion criteria may apply. Key

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
Change of frequency of packed RBC transfusions.	From 6 months before recruitment to 104 weeks post-transplant
Change of proportion of HbF/Hb.	within 104 weeks post-transplant
Change of volume of packed RBC transfusions.	From 6 months before recruitment to 104 weeks post-transplant
Change in total hemoglobin from baseline.	within 104 weeks post-transplant
Change of HbF from baseline.	within 104 weeks post-transplant

Countries

China

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026