Condition: Transfusion-dependent α-Thalassemia · Sponsor: First Affiliated Hospital of Guangxi Medical University
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 \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].
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| The affiliated hospital of guangxi medical university | Nanning, Guangxi, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05757245