drugset / Trial / NCT03276455
Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified
Phase 1/2
Unknown
10 enrolled
Nanfang Hospital, Southern Medical University
Guangdong Yike Gene Science and Technology CO.,Ltd · collab
NaSingle-groupOpen-labelTreatment
Summary
This is a single group, open label study in 10 subjects who are 8 years of age or older with beta-thalassemia major. The objective of this study is to evaluate the safety and efficacy of autologous hematopoietic stem cell transduced with lentiviral vector for the treatment of beta-thalassemia major.
Timeline
- Start
- 2017-09-15
- Primary completion
- 2020-09-15
- Completion
- 2021-09-15
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Autologous CD34+ cells genetically modified | Cell therapy | 3e+06 cells/kg | Other |