A phase 3 trial in participants with obesity due to Bardet-Biedl syndrome or Alström syndrome: efficacy and safety of the melanocortin 4 receptor agonist setmelanotide

Abstract:

Introduction: This randomized Phase 3 trial evaluated the effect of setmelanotide, a melanocortin 4 receptor agonist, on weight loss, hunger reduction, and safety outcomes in individuals (aged ≥6 years) with obesity and a genetically confirmed diagnosis of Bardet-Biedl syndrome (BBS) or Alström syndrome (AS), conditions believed to disrupt hypothalamic leptin-melanocortin signaling. Methods: For inclusion, obesity was defined as body mass index ≥30 kg/m2 (in those aged ≥16 years) or weight >97th percentile (in those aged 6–15 years). Individuals were randomized and received setmelanotide or placebo for 14 weeks, followed by open-label setmelanotide so that all participants received at least 1 year of drug. Body weight, height, hunger scores, and treatment-emergent adverse events (AEs) were assessed. The primary endpoint was the proportion of participants (≥12 years) who achieved ≥10 …

Año de publicación:

2021

Keywords:

Fuente:

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