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

Phase 2

Recruiting

• Conditions: Prader-Willi Syndrome; Obesity; Hyperphagia
• Interventions: Drug: Setmelanotide

• Registration Number: NCT06772597
• Lead Sponsor: Rhythm Pharmaceuticals, Inc.

Brief Summary

This is a Phase 2, single-center, 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 26 weeks.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-01-10
• Study First Submitted QC: 2025-01-10
• Study First Posted: 2025-01-13
• Status Verified: 2025-03
• Study Start: 2025-03-04
• Last Update Submitted: 2025-03-18
• Last Update Posted: 2025-03-19
• Primary Completion: 2026-01-31
• Study Completion: 2026-01-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Setmelanotide (Open-label)	Setmelanotide	-

Primary Outcome Measures

Name	Time	Method
Frequency and severity of adverse events (AEs)	Baseline to Week 26

Secondary Outcome Measures

Name	Time	Method
Proportion of patients who achieve 5% reduction in BMI	Baseline to Week 26
Mean percent change in BMI	Baseline to Week 26
Mean percent change in BMI and weight in patients ≥18 years of age	Baseline to Week 26
Mean change in BMI Z-Score and BMI % of 95th percentile	Baseline to Week 26
Proportion of patients who achieved 5% reduction in weight from Baseline in patients ≥18 years of age and who achieved ≥0.2 reduction in BMI Z-score in patients <18 years of age	Baseline to Week 26
Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score	Baseline to Week 26

Trial Locations

Locations (1)

University of Florida; 🇺🇸 Gainesville, Florida, United States