drugset / Trial / NCT04211480
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
Completed
6 enrolled
Bioray Laboratories
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army · collabXiangya Hospital of Central South University · collab
NaSingle-groupOpen-labelTreatment
Summary
This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.
Timeline
- Start
- 2020-10-01
- Primary completion
- 2023-10-15
- Completion
- 2023-11-27
Publications
- Fu B, Liao J, Chen S, Li W, Wang Q, Hu J, Yang F, Hsiao S, Jiang Y, Wang L, Chen F, Zhang Y, Wang X, Li D, Liu M, Wu Y. CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric beta0/beta0 transfusion-dependent beta-thalassemia. Nat Med. 2022 Aug;28(8):1573-1580. doi: 10.1038/s41591-022-01906-z. Epub 2022 Aug 4.
- Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | γ-globin reactivated autologous hematopoietic stem cells | Cell therapy | — | — |