NCT06655662

Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.

An Open, Multi-center, Phase I Clinical Study on the Safety and Efficacy of HGI-001 Injection in Patients with Transfusion-Dependent Β-Thalassemia.

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Shenzhen Hemogen · Industry
• Sex: All
• Age: 6 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia. The study aims to evaluate the safety and efficacy of autologous lentiviral-transduced CD34+ human hematopoietic stem cells (hHSCs) using the gene therapy product HGI-001.

Detailed description

The investigators will recruit transfusion-dependent β-thalassaemia patients and collect their autologous hematopoietic stem cells, which will be modified with the LentiHBBT87Q system to restore β-globin expression. After conditioning, the autologous hematopoietic stem cells with restored β-globin will be reinfused to the patients and followed up for two years to collect data.

Conditions

• Β-thalassemia

Interventions

Type	Name	Description
OTHER	β-globin restored autologous hematopoietic stem cells	Eight transfusion-dependent β-thalassaemia subjects aged 6-35 years will be reinfused with β-globin restored autologous hematopoietic stem cells modified with LentiHBBT87Q

Timeline

• Start date: 2024-06-12
• Primary completion: 2025-12-31
• Completion: 2026-12-31
• First posted: 2024-10-23
• Last updated: 2024-10-23

Locations

3 sites across 1 country: China