Open-Label Extension Study of Setmelanotide

This is an open-label extension study designed to evaluate the long-term safety and tolerability of continued setmelanotide treatment in male and female patients ≥2 years of age who have completed or transitioned from a previous study with setmelanotide for rare genetic, syndromic, or acquired diseases of obesity upstream of the MC4R pathway.

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Key information

Conditions

Obesity Associated With Defects in Leptin-melanocortin Pathway Body Weight Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Sex Development Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Gonadal Disorders Gonadal Dysgenesis Hypogonadism Male Urogenital Diseases Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight Pathological Conditions, Signs and Symptoms Sexual Infantilism Signs and Symptoms Urogenital Abnormalities Urogenital Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

HonorHealth Research Institute, Scottsdale, Arizona, United States

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

Males and females ≥2 years of age who have completed a previous setmelanotide clinical study and who have a condition or indication for which Rhythm plans further clinical development.
Agree to use a highly effective form of contraception and follow the study contraception requirements throughout study duration and for 90 days after.
Demonstrated clinical benefit in the previous setmelanotide study, as determined by the Investigator.

Key Exclusion Criteria:

Any gene variant, syndromic, or acquired disease for which setmelanotide is currently approved in the United States (i.e., patients eligible for commercial setmelanotide (IMCIVREE®).
Any new or worsening depression resulting in suicidal thoughts and/or behaviors.
Discontinuation from a previous setmelanotide study.
History or close family history (parents or siblings) of melanoma.
Pregnant and/or breastfeeding women

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

Setmelanotide

Drug

Open-label daily injections of setmelanotide

Other names: RM-493, Imcivree

Primary outcomes

Safety and tolerability of setmelanotide assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: Up to 3 years

Sponsors and collaborators

Lead sponsor

Rhythm Pharmaceuticals, Inc.

Industry

Registry information

Official study title

Open-Label Extension Study of Setmelanotide in Patients With a Rare Genetic, Syndromic or Acquired Disease of the MC4R Pathway

Important dates

Study start: 2024
Primary completion: 2026
Study completion: 2026
First posted: Sep 19, 2024
Registry last updated: Jul 15, 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.

Published trials that share one or more normalized conditions with this study.

Completed