NCT05757245

A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants

A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With Transfusion-dependent α-Thalassemia by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral Vector (GMCN-508A Drug Product)

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: First Affiliated Hospital of Guangxi Medical University · Academic / Other
• Sex: All
• Age: 5 Years – 35 Years
• Healthy volunteers: Not accepted

Summary

This is a non-randomized, open label, single-site, single-dose, phase 1 study in up to 5 participants (between 5 and 35 years of age, inclusive) with Transfusion-dependent α-thalassemia. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using GMCN-508A Drug Product \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].

Detailed description

Subject participation for this study will be 5 years.

Conditions

• Transfusion-dependent α-Thalassemia

Interventions

Type	Name	Description
GENETIC	GMCN-508A Drug Product	Transplant of autologous hematopoietic stem and progenitor cells transduced with GMCN-508A lentiviral vector.

Timeline

• Start date: 2023-05-08
• Primary completion: 2028-08-01
• Completion: 2030-12-31
• First posted: 2023-03-07
• Last updated: 2023-04-18

Locations

1 site across 1 country: China