A Study of Setmelanotide in Patients With Prader-Willi Syndrome

NCTNCT06772597Other idRM-493-043

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Active, not recruiting
Phase: Phase 2
Enrollment: 18 participants (actual)
Sponsor: Rhythm Pharmaceuticals, Inc. · Industry
Design: Allocation not applicable · Open label (no masking)
Countries: 1 country
United States
Start: 4 Mar 2025
Primary completion: 31 Oct 2026 (estimated)
Completion: 31 Oct 2027 (estimated)
First posted: 13 Jan 2025
Last update posted: 12 Feb 2026
Conditions: Prader-Willi Syndrome · Obesity · Hyperphagia

Timeline

1 trial · 1 ongoing

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

2025202620272028

Ph 2
A Study of Setmelanotide in Patients With Prader-Willi Syndrome
NCT06772597
4 Mar 2025 to est. 31 Oct 2027

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

SetmelanotideDrug

Arms1

Experimental
Setmelanotide (Open-label)

Primary outcomes1

Outcomes the sponsor listed as primary.

Frequency and severity of adverse events (AEs)
Time frame · Baseline to Week 52

Secondary outcomes(6)

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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: All
Age: from 6 Years up to 65 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

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.

PepBank is a research reference. Talk to a doctor before you use any peptide.