Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
CRISPR-Cas9 β-globin Restoration for CVS-654 β-thalassemia
NCT04205435Phase 2Terminated
- Sponsor
- BRL Medicine Inc.
- Start
- Nov 2021
- Primary completion
- Jun 2022
- Modality
- Gene Editing, Gene Silencing, Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of LentiHBBT87Q in beta thalassemia major, sponsored by BRL Medicine Inc. Primary endpoints: Proportion of subjects with engraftment at 42 days and Incidence and severity of adverse events at 60 days. Started Nov 2021; status: terminated.
02
Drugs
LentiHBBT87Q
03
Conditions
beta thalassemia major
Beyond the preview
Go deeper on NCT04205435.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NCT04205435.