DRUG
Setmelanotide
Setmelanotide (daily subcutaneous injection)
Status
Active, not recruiting
Phase
Phase 2
Enrollment
18
Locations
1
Results
Not posted
Publications
0
Study 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.
Interventions
DRUG
Setmelanotide (daily subcutaneous injection)
Timeline
First posted
Jan 13, 2025
Study start
Mar 4, 2025
Primary completion
Oct 31, 2026
Study completion
Oct 31, 2027
Results posted
Not reported
Registry updated
Feb 12, 2026
Outcomes
Frequency and severity of adverse events (AEs)
Time frame · Baseline to Week 52
Proportion of patients who achieve 5% reduction in BMI
Time frame · Baseline to Week 52
Mean percent change in BMI
Time frame · Baseline to Week 52
Mean percent change in BMI and weight in patients ≥18 years of age
Time frame · Baseline to Week 52
Mean change in BMI Z-Score and BMI % of 95th percentile
Time frame · Baseline to Week 52
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
Time frame · Baseline to Week 52
Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score
Time frame · Baseline to Week 52
Eligibility
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.
Study locations
United States. Showing up to 24 locations stored in the fast local snapshot.
University of Florida
Gainesville, Florida, United States
Publications
No PMID-linked publications were present in this registry snapshot.
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