A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants
Recruiting
Phase 1
Interventional Study
Transfusion-dependent α-Thalassemia
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 5 – 35
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 5 (estimated)
- Sponsor
- First Affiliated Hospital of Guangxi Medical University · Other
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About This Trial
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 lenti…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. The subject himself/herself or one legal guardian/agent of the subject is required to fully understand the study and voluntarily sign a written informed consent.
2. Ages 5 to 35, no gender limitation.
3. The clinical diagnosis of Transfusion-dependent α-Thalassemia.Transfusio…
Contacts
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