A Study of Setmelanotide in Patients With Prader-Willi Syndrome

NCT06772597 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: RM-493-043
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Prader-Willi Syndrome; Obesity; Hyperphagia

Keywords

melanocortin 4 receptor (MC4R), MC4R agonist

Outcome Measures

Primary Outcomes (1)

• Frequency and severity of adverse events (AEs)

  Baseline to Week 52

Secondary Outcomes (6)

• Proportion of patients who achieve 5% reduction in BMI

  Baseline to Week 52

• Mean percent change in BMI

  Baseline to Week 52

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

  Baseline to Week 52

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

  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

  Baseline to Week 52

Study Arms (1)

Setmelanotide (Open-label)

EXPERIMENTAL

Drug: Setmelanotide

Interventions

Setmelanotide DRUG

Setmelanotide (daily subcutaneous injection)

Also known as: Imcivree

Setmelanotide (Open-label)

Eligibility Criteria

• Age: 6 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Rhythm Pharmaceuticals, Inc.: lead

Study Sites (1)

University of Florida

Gainesville, Florida, 32610-0296, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome; Obesity; Hyperphagia

Interventions

setmelanotide

Condition Hierarchy (Ancestors)

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

Study Officials

• David Meeker, MD

  Rhythm Pharmaceuticals, Inc.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 10, 2025
• First Posted: January 13, 2025
• Study Start: March 4, 2025
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): October 31, 2027
• Last Updated: February 12, 2026

Record last verified: 2026-02

Locations

US (1)