Last updated on November 2019

Setmelanotide (RM-493) Melanocortin-4 Receptor (MC4R) Agonist in Bardet-Biedl Syndrome (BBS) and Alstr m Syndrome (AS) Patients With Moderate to Severe Obesity


Brief description of study

This pivotal, phase 3 study is designed to confirm the efficacy and safety of setmelanotide, a potent melanocortin receptor type 4 (MC4R) agonist, for the treatment of obesity and hyperphagia in patients with Bardet Biedl syndrome (BBS) or Alstrm syndrome (AS). The study's primary efficacy endpoint will evaluate the proportion of patients (12 years of age at baseline) who lose 10% of their baseline body weight following approximately (~) 52 weeks of treatment with setmelanotide compared to a historical control rate. The study will consist of 3 treatment periods. Eligible patients will enter a 14 week, randomized, double-blind, placebo-controlled treatment period (Period 1) that will be followed by a 38 week open label treatment period (Period 2) in which all patients will receive setmelanotide. The primary analysis will be performed after Period 2. Following Period 2, patients will continue open-label treatment for 14 weeks (Period 3) after which they may be enrolled into a separate treatment extension study.

Clinical Study Identifier: NCT03746522

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