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NCT Number: NCT06772597

A 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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Body Weight Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hyperphagia Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight Pathological Conditions, Signs and Symptoms Signs and Symptoms Signs and Symptoms, Digestive

Age range

6 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of Florida

Gainesville, Florida, 32610-0296, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Setmelanotide

Drug

Setmelanotide (daily subcutaneous injection)

Other names: Imcivree

Primary outcomes

  1. Frequency and severity of adverse events (AEs)

    Time frame: Baseline to Week 52

Secondary outcomes

  1. Proportion of patients who achieve 5% reduction in BMI

    Time frame: Baseline to Week 52

  2. Mean percent change in BMI

    Time frame: Baseline to Week 52

  3. Mean percent change in BMI and weight in patients ≥18 years of age

    Time frame: Baseline to Week 52

  4. Mean change in BMI Z-Score and BMI % of 95th percentile

    Time frame: Baseline to Week 52

  5. 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

  6. Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score

    Time frame: Baseline to Week 52

Sponsors and collaborators

Lead sponsor

Rhythm Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2026
Study completion
2027
First posted
Jan 13, 2025
Registry last updated
Feb 12, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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