Clinical Trials Directory

Trials / Completed

CompletedNCT03746522

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

A Phase 3 Trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity

Status
Completed
Phase
Phase 3
Study type
Interventional
Enrollment
52 (actual)
Sponsor
Rhythm Pharmaceuticals, Inc. · Industry
Sex
All
Age
6 Years
Healthy volunteers
Not accepted

Summary

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 participants with Bardet Biedl syndrome (BBS) or Alström syndrome (AS). The study's primary efficacy endpoint is to evaluate the proportion of participants (≥ 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.

Detailed description

Eligible participants 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 participants will receive setmelanotide. Following Period 2, participants will continue receiving open-label setmelanotide for 14 weeks (Period 3), after which they could enroll into a separate treatment extension study.

Conditions

Interventions

TypeNameDescription
DRUGSetmelanotideSC injection of setmelanotide
DRUGPlaceboSC injection of placebo

Timeline

Start date
2018-11-23
Primary completion
2020-11-16
Completion
2021-03-08
First posted
2018-11-19
Last updated
2023-12-01
Results posted
2023-12-01

Locations

12 sites across 6 countries: United States, Canada, France, Puerto Rico, Spain, United Kingdom

Regulatory

Source: ClinicalTrials.gov record NCT03746522. Inclusion in this directory is not an endorsement.