Engasertib versus Placebo for Bleeding in Hereditary Hemorrhagic Telangiectasia
The safety profile of engasertib was similar to that of placebo except for mild-to-moderate rash, which resolved in most patients who continued to receive the drug. Engasertib treatment was associated with decreases in epistaxis frequency and duration. (Funded by Vaderis Therapeutics; ClinicalTrials.gov number, NCT05406362.).
Authors
- Corrado Bernasconi (ORCID: https://orcid.org/0000-0002-1019-5468)
- Sophie Dupuis‐Girod (ORCID: https://orcid.org/0000-0002-8834-5526)
- Antoni Riera‐Mestre (ORCID: https://orcid.org/0000-0001-9411-804X)
- Debra Barker (ORCID: https://orcid.org/0009-0000-3626-9376)
- Hedvika Lazar
- Vicente Gómez del Olmo (ORCID: https://orcid.org/0000-0002-7992-3769)
- Roberto Bertè (ORCID: https://orcid.org/0000-0002-3978-1380)
- Benedict Nwogoh (ORCID: https://orcid.org/0000-0002-2551-5647)
- Elisabetta Buscarini (ORCID: https://orcid.org/0000-0003-0863-0624)
- Josefien Hessels (ORCID: https://orcid.org/0000-0002-9001-9893)
- Thibaut Van Zele (ORCID: https://orcid.org/0000-0002-6031-4293)
- Hanny T. Al-Samkari (ORCID: https://orcid.org/0000-0001-6175-1383)
- Pierre Saint-Mézard
- Raquel Torres-Iglesias (ORCID: https://orcid.org/0000-0001-9204-9888)
- D Picard (ORCID: https://orcid.org/0009-0005-9078-4535)
- Pamela G Hodges
- Hans‐Jurgen Mager
Institutions
- Inserm (FR)
- Commissariat à l'Énergie Atomique et aux Énergies Alternatives (FR)
- Ghent University Hospital (BE)
- Bellvitge University Hospital (ES)
- Hôpital Femme Mère Enfant (FR)
- Institut d'Investigació Biomédica de Bellvitge (ES)
- Massachusetts General Hospital (US)
- St. Antonius Ziekenhuis (NL)
- NBE Therapeutics (Switzerland) (CH)
- Instituto Ramón y Cajal de Investigación Sanitaria (ES)
- Azienda Ospedaliera Ospedale Maggiore (IT)
- CRISPR Therapeutics (Switzerland) (CH)
- PIQUR Therapeutics (Switzerland) (CH)
- Université Grenoble Alpes (FR)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-11-26
- DOI
- https://doi.org/10.1056/nejmoa2504411
- Citations
- 2
- Primary Topic
- Vascular Anomalies and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 3.25