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

December 10, 2018
Diabetes, Obesity, Pediatrics
Rushika Conroy, MD
Baystate Children’s Specialty Center, 50 Wason Avenue, Springfield, MA
Rhythm Pharmaceuticals, Inc. developed the trial drug setmelanotide, a MSH messenger substance, which is hoped to replace the messenger substances that are missing in someone with BBS or ALMS1 gene variation. Setmelanotide is an investigational drug, meaning it has not yet been approved for use outside of clinical trials.
• BBS clinical diagnosis or AS diagnosis
• Over the age of 12
• Obese (BMI ≥30 kg/m2 for patients ≥16 years of age or weight >97th percentile for age and sex on growth chart assessment for patients 12 to 15 years of age)
Interventional (Clinical Trial)
Victoria Cobb