Using setmelanotide to treat obesity in patients with Prader-Willi Syndrome
A Phase 2, Single-center, Open-label Study of Setmelanotide in Patients With Prader-Willi Syndrome
This study is testing if a new medication called setmelanotide can help people with Prader-Willi Syndrome manage their weight and improve related symptoms.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 6 Years to 65 Years |
| Sex | All |
| Sponsor | Rhythm Pharmaceuticals, Inc. Industry-sponsored |
| Locations | 1 site (Gainesville, Florida) |
| Trial ID | NCT06772597 on ClinicalTrials.gov |
What this trial studies
This Phase 2, single-center, open-label study aims to evaluate the safety and efficacy of setmelanotide in individuals aged 6 to 65 with obesity due to Prader-Willi Syndrome (PWS). The study plans to enroll up to 20 patients who will receive a daily subcutaneous dose of setmelanotide for a duration of up to 26 weeks. The primary focus is on assessing the impact of this treatment on weight management and related symptoms in this specific patient population.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged 6 to 65 with a confirmed diagnosis of Prader-Willi Syndrome and obesity.
Not a fit: Patients with significant comorbidities or those currently using weight-modulating medications may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve weight management and reduce hyperphagia in patients with Prader-Willi Syndrome.
How similar studies have performed: While this approach is novel in the context of Prader-Willi Syndrome, similar studies using melanocortin receptor agonists have shown promise in other obesity-related conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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 HbA1c, eGFR, ALT, AST, bilirubin, T4 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.
Where this trial is running
Gainesville, Florida
- University of Florida — Gainesville, Florida, United States (Recruiting)
Study contacts
- Study coordinator: Rhythm Clinical Trials
- Email: clinicaltrials@rhythmtx.com
- Phone: (857) 264-4280
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.