ELLIPSE Study: A Phase 1 study evaluating the tolerance of bevacizumab nasal spray in the treatment of epistaxis in hereditary hemorrhagic telangiectasia.

Abstract : Background: Hereditary hemorrhagic telangiectasia (HHT) is a dominantly inherited genetic vascular disorder in which epistaxis is the most frequent manifestation, responsible for high morbidity. Management of this symptom has no standard, and local treatments are often aggressive. Their efficacy is variable and has not been proven. Anti-angiogenic drugs, such as bevacizumab, are a new treatment strategy. Its systemic administration in patients with HHT improves liver damage-related symptoms and epistaxis. To limit the systemic adverse effects of bevacizumab and to ease administration, a local administration seems suitable. Primary objective: To evaluate the tolerance of increasing doses of bevacizumab administered as a nasal spray in patients with HHT-related epistaxis. Secondary objectives were to study the bioavailability and efficacy of bevacizumab against epistaxis when given as a nasal spray. Methodology: Phase 1, randomized, double-blind, placebo-controlled, monocentric study performed sequentially (dose escalation) on 5 groups of 8 patients. Each group was made up of 6 verum and 2 placebos. Five increasing doses of bevacizumab nasal spray (25 mg/mL) were evaluated: 12.5, 25, 50, 75 and 100 mg. Results: A total of 40 patients were included between October 2011 and October 2012. Bevacizumab nasal spray was well tolerated in all patients and the drug was not detected in their serum. No dose limiting toxicity was observed. No efficacy was observed at any dose in this study. Conclusion: Based on these results, bevacizumab nasal spray is a safe treatment of epistaxis in HHT. However, a randomized Phase 2 study is needed to determine its efficacy. Trial Registration: ClinicalTrials.gov Identifier #NCT01507480.
Type de document :
Article dans une revue
mAbs, Taylor & Francis, 2014, 6 (3), pp.793-8. 〈10.4161/mabs.28025〉
Liste complète des métadonnées

Littérature citée [15 références]  Voir  Masquer  Télécharger

https://hal-univ-tours.archives-ouvertes.fr/hal-00991138
Contributeur : Gilles Paintaud <>
Soumis le : mercredi 14 mai 2014 - 18:33:32
Dernière modification le : vendredi 22 juin 2018 - 17:08:05
Document(s) archivé(s) le : jeudi 14 août 2014 - 12:05:53

Fichier

Ellipse12112013.pdf
Fichiers produits par l'(les) auteur(s)

Identifiants

Collections

Citation

S. Dupuis-Girod, A. Ambrun, E. Decullier, G. Samson, A. Roux, et al.. ELLIPSE Study: A Phase 1 study evaluating the tolerance of bevacizumab nasal spray in the treatment of epistaxis in hereditary hemorrhagic telangiectasia.. mAbs, Taylor & Francis, 2014, 6 (3), pp.793-8. 〈10.4161/mabs.28025〉. 〈hal-00991138〉

Partager

Métriques

Consultations de la notice

261

Téléchargements de fichiers

255