A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia

EdiGene

Status and phase: Terminated; Phase 1

Conditions: Transfusion Dependent Beta-Thalassaemia

Treatments: Biological: ET-01

Study type: Interventional

Funder types: Industry

Identifiers

NCT04925206

EDG-001

Details and patient eligibility

About

This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.

Enrollment: 8 patients

Sex: All

Ages: 12 to 35 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Subjects and/or legal representative fully understand and voluntarily sign informed consent forms.
• Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol.
• Be not appropriate to receive conventional allogeneic hematopoietic stem cell transplantation.
• Lansky/Karnofsky score ≥ 70%.
• Eligible for hematopoietic stem cell transplantation and conditioning with busulfan as per investigator's judgement.

Other protocol defined inclusion criteria may apply.

Key Exclusion Criteria:

• Subjects with associated α-thalassemia.
• Subjects with any clinically significant acute or uncontrolled infections.
• History of uncontrolled epilepsy or other mental disorders.
• Previous treatment with allogeneic bone marrow transplantation or gene therapy.

Other protocol defined exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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8 participants in 1 patient group

ET-01

Experimental group

Treatment:

Biological: ET-01