Setmelanotide - Daily vs weekly with crossover to open label
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) with a Crossover to Open-Label Once Weekly Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation
IRAS ID
1004467
Contact name
Madhura Srinivasan
Contact email
Sponsor organisation
Rhythm Pharmaceuticals, Inc.
Eudract number
2021-004597-65
Clinicaltrials.gov Identifier
Research summary
This is a study to assess the efficacy of a once daily formulation of Setmelanotide versus a once weekly formulation over a 13 week administration period for patients with genetic disorders of obesity (BBS, POMC, PCSK1, LEPR or heterozygous PPL). These genetic disorders result in obesity related comorbidities such as type 2 diabetes. Stemelanotide can help to moderate appetite and therefore control weight to reduce the risk of life limiting comorbidities. Patients who have already been taking part in the RM-493-033 extension study for 6 months will be eligible to take part in this study. This will be a multi-centre trial taking place across the United States, Canada and Europe; 30 patients will be recruited from the following age groups: 20 patients aged ≥18 years, 6 aged ≥12 to <18 years and 4 ages ≥6 to <12. The screening period of up to 21 days will allow for transition from the long term extension study, a week long run-in period will follow to ensure continuation of the participants current dose level for daily Setmelanotide. Participants will then be randomised to either the daily dose or weekly dose arm (this will be double blind) for 13 weeks of treatment, this is followed by a 13 week open label phase where all participants will receive a weekly dose. Finally, a 4 weeks follow up period will have participants return to their daily run-in dose in preparation for returning to the long term extension study.
REC name
London - Riverside Research Ethics Committee
REC reference
22/LO/0264
Date of REC Opinion
19 May 2022
REC opinion
Further Information Favourable Opinion