AZP-531, an unacylated ghrelin analog, improves food-related behavior in patients with Prader-Willi syndrome : A randomized placebo-controlled trial
Allas, Soraya (Alizé Pharma, Ecully, France)
Caixàs i Pedragós, Assumpta (Parc Taulí Hospital Universitari. Institut d'Investigació i Innovació Parc Taulí (I3PT))
Poitou, Christine (Hôpital Pitié-Salpêtrière, Paris)
Coupaye, Muriel (Hôpital Pitié-Salpêtrière, Paris)
Thuilleaux, Denise (Hôpital Marin de Hendaye)
Lorenzini, Françoise (Hôpital Rangueil, Toulouse)
Diene, Gwenaëlle (Hôpital des Enfants, Toulouse)
Crinò, Antonino (Ospedale Pediatrico Bambino Gesù, Rome)
Illouz, Frédéric (Centre Hospitalier Universitaire Angers)
Grugni, Graziano (Istituto Auxologico Italiano Piancavallo)
Potvin, Diane (Excelsus Statistics, Montréal)
Bocchini, Sarah (Ospedale Pediatrico Bambino Gesù, Rome)
Delale, Thomas (Alizé Pharma, Ecully, France)
Abribat, Thierry (Alizé Pharma, Ecully, France)
Tauber, Maithé (Université Paul Sabatier, Toulouse)
Universitat Autònoma de Barcelona

Date:	2018
Abstract:	Prader-Willi syndrome (PWS) is characterized by early-onset hyperphagia and increased circulating levels of the orexigenic Acylated Ghrelin (AG) hormone with a relative deficit of Unacylated Ghrelin (UAG). AZP-531, a first-in-class UAG analog, was shown to inhibit the orexigenic effect of AG in animals, to improve glycemic control and decrease body weight in humans. We aimed to investigate the safety and efficacy of AZP-531 in patients with PWS for whom no approved treatment for hyperphagia is currently available. Multi-center, randomized, double-blind, placebo-controlled trial. Forty-seven patients with genetically confirmed PWS and evidence of hyperphagia received daily subcutaneous injections of AZP-531 (3 and 4 mg for 50-70 kg and.
Note:	Altres ajuts: This study was supported by Alizé Pharma, sponsor of the trial (EudraCT: 2014- 001670-34). The sponsor Alizé Pharma was involved in the design of the study, data collection and analysis, decision to publish, and preparation of the manuscript.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Published in:	PloS one, Vol. 13 (january 2018) , ISSN 1932-6203

DOI: 10.1371/journal.pone.0190849
PMID: 29320575

19 p, 2.0 MB

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Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Parc Taulí Research and Innovation Institute (I3PT
Articles > Research articles
Articles > Published articles