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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

NA

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-18

Study Completion Date

2025-03-12

Brief Summary

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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.

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Detailed Description

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Conditions

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Transfusion Dependent Beta-Thalassaemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ET-01

BCL11A Enhancer modified Autologous Hematopoietic Stem Cells

Group Type EXPERIMENTAL

ET-01

Intervention Type BIOLOGICAL

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

Interventions

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ET-01

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.

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.

Minimum Eligible Age

6 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No