β-globin Restored Autologous HSC in β-thalassemia Major Patients

NCT04205435 ↗

Phase 1/2 Terminated 2 enrolled Bioray Laboratories The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army · collab

NaSingle-groupOpen-labelTreatment

Summary

This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.

Timeline

Start: 2021-11-01
Primary completion: 2022-06-01
Completion: 2022-07-25

Outcome

Outcome not reported

Stopped (Business): “Sponsor decision”

Publications

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	β-globin restored autologous HSC	CRISPR / gene editing	—	—