This is a Phase 2, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age. Up to 20 patients are planned to be enrolled. Patients will take a daily subcutaneous dose of open-label setmelanotide for up to 52 weeks.
Eligibility
Sex
ALL
Min age
6 Years
Max age
65 Years
Healthy volunteers
No
Key Inclusion Criteria:
* Confirmed diagnosis of Prader-Willi Syndrome (PWS)
* Age 6 to 65
* BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th Percentile for age and sex
* Agree to use a highly effective form of contraception and follow the study contraception requirements throughout study duration and for 90 days after.
* Patient and/or guardian is able to communicate well with the Investigator, understand and comply with the requirements of the study, and understand English and sign the written informed consent.
Exclusion Criteria:
* Use of weight modulating medications
* Abnormal eGFR, ALT, AST, and bilirubin values
* Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results.
* Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose
* Hypersensitivity to setmelanotide
* Diagnosis of severe psychiatric disorders
* Pregnant and/or breastfeeding
Other protocol defined Inclusion/Exclusion criteria may apply.
Primary outcome measure(s)
Frequency and severity of adverse events (AEs) — Baseline to Week 52
Trial sites (1)
Facility
City
Region
Status
University of Florida
Gainesville
Florida
More Rhythm Pharmaceuticals, Inc. trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time on our Cookie Policy page. See also our Privacy Policy.