A Study of Setmelanotide in Patients With Prader-Willi Syndrome

February 10, 2026 updated by: Rhythm Pharmaceuticals, Inc.

A Phase 2 Open-label Study of Setmelanotide in Patients With Prader-Willi Syndrome

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

18

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Gainesville, Florida, United States, 32610-0296
        • University of Florida

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Setmelanotide (Open-label)
Drug: Setmelanotide
Setmelanotide (daily subcutaneous injection)
Other Names:
  • Imcivree

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Frequency and severity of adverse events (AEs)
Time Frame: Baseline to Week 52
Baseline to Week 52

Secondary Outcome Measures

Outcome Measure
Time Frame
Proportion of patients who achieve 5% reduction in BMI
Time Frame: Baseline to Week 52
Baseline to Week 52
Mean percent change in BMI
Time Frame: Baseline to Week 52
Baseline to Week 52
Mean percent change in BMI and weight in patients ≥18 years of age
Time Frame: Baseline to Week 52
Baseline to Week 52
Mean change in BMI Z-Score and BMI % of 95th percentile
Time Frame: Baseline to Week 52
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
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
Baseline to Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Rhythm Pharmaceuticals, Inc.

Investigators

  • Study Chair: David Meeker, MD, Rhythm Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 4, 2025

Primary Completion (Estimated)

October 31, 2026

Study Completion (Estimated)

October 31, 2027

Study Registration Dates

First Submitted

January 10, 2025

First Submitted That Met QC Criteria

January 10, 2025

First Posted (Actual)

January 13, 2025

Study Record Updates

Last Update Posted (Actual)

February 12, 2026

Last Update Submitted That Met QC Criteria

February 10, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RM-493-043

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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