Health Trials

Transfusion-dependent α-Thalassemia

1

Review clinical trials related to Transfusion-dependent α-Thalassemia. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

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 \[autologous CD34+ hematopoietic stem cells transduced with GMCN-508A lentiviral vector encoding the human α-globin gene\].

Participants needed: 5
Trial details
Phase: Phase 1Age: 5-35Biological sex: AllType: InterventionalSponsor: First Affiliated Hospital of Guangxi Medical UniversityUpdated: Apr 18, 2023Locations: 1
Eligibility criteria

The subject himself/herself or one legal guardian/agent of the subject is requir... [+6]

Diagnosed with mild α-thalassemia, Hb Bart's edema, ATRx α-thalassemia, hemoglob... [+12]

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