Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Phase 3 single-dose CTX001 study in pediatric transfusion-dependent beta-thalassemia
2021-002172-39Phase 3Active
- Sponsor
- Vertex Pharmaceuticals
- Start
- Sep 2021
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in beta thalassemia, sponsored by Vertex Pharmaceuticals. Primary endpoint: Proportion of subjects achieving TI12. Started Sep 2021; status: active.
02
Drugs
exagamglogene autotemcel
03
Conditions
beta thalassemia
Beyond the preview
Go deeper on 2021-002172-39.
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 2021-002172-39.