Clinical Trial

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

Recruiting Phase 1
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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 \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2023-02-24.
Status change: Not Yet Recruiting → Recruiting 2023-04-16
Trial Details
NCT Number NCT05757245
Lead Sponsor First Affiliated Hospital of Guangxi Medical University
Collaborators: Genmedicn Biopharma Ltd.
Conditions Transfusion-dependent α-Thalassemia
Enrollment 5 participants
Start Date 2023-05-08
Primary Completion 2028-08-01 (estimated)
Study Completion 2030-12-31 (estimated)
Updated on ClinicalTrials.gov 2023-04-18