A Single-center Study of Setmelanotide in Patients With Prader-Willi Syndrome

Launched by RHYTHM PHARMACEUTICALS, INC. · Jan 10, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called setmelanotide to see how safe and effective it is for people with Prader-Willi Syndrome (PWS), a condition that often leads to obesity and extreme hunger. The trial will include up to 20 participants between the ages of 6 and 65 who have been diagnosed with PWS. To qualify for the study, participants should have a body mass index (BMI) that indicates obesity, and they must agree to use effective birth control during the study.

During the trial, participants will receive a daily injection of setmelanotide for up to 26 weeks. The study is currently recruiting, and it’s important that participants or their guardians can communicate well and understand the study requirements. However, those with certain health issues, recent participation in other clinical trials, or specific allergies may not be eligible. This trial offers a unique opportunity to potentially help improve treatment options for individuals living with PWS.

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Eligibility 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.