Trials / NCT03746522
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
Completed Phase 3 Drug / neuroprotection
- Sponsor
- Rhythm Pharmaceuticals, Inc.
- Treatment
- Setmelanotide; Placebo
- Gene
- Not stated
- Conditions
- Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
- Ages
- 6 Years to no upper limit
- Enrollment
- 52 participants
- Started
- 2018-11-23
- Main results expected
- 2020-11-16
- Results posted
- Yes
- Record last updated
- 2023-12-01
What the study is testing
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.
Where it runs
Canada (1)
- Alberta Health Services, Edmonton, Alberta
France (2)
- Sorbonne University, Hôpital de la Pitié-Salpêtrière, Paris
- Centre Hospitalier Universitaire de Strasbourg, Strasbourg
Puerto Rico (1)
- UPR Medical Sciences Campus, Rio Piedras
Spain (1)
- Universidad Autónoma de Madrid University Hospital Niño, Madrid
United Kingdom (1)
- St. Thomas Hospital, London
United States (6)
- Wr-McCr, Llc, San Diego, California
- UMMS Baystate Health; BAYSTATE MEDICAL CENTER; Baystate Children's Specialty Center, Springfield, Massachusetts
- Columbia University Center, New York, New York
- M3 Wake Research, Raleigh, North Carolina
- University of Tennessee Health Science Center, Memphis, Tennessee
- Marshfield Clinic Research Foundation, Marshfield, Wisconsin