Active — Not RecruitingPhase 2

A Study of Setmelanotide in Patients With Prader-Willi Syndrome

United States18 participantsStarted 2025-03-04

Plain-language summary

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.

Who can participate

Age range

6 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Frequency and severity of adverse events (AEs)

Timeframe: Baseline to Week 52

Trial details

NCT IDNCT06772597

SponsorRhythm Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-10-31

View on ClinicalTrials.gov More Prader-Willi Syndrome trials