Open-Label Extension Study of Setmelanotide

NCT06596135 | Enrolling By Invitation Phase 3 FDA Drug

• 1 other identifier: RM-493-042
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 13

Brief Summary

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.

Conditions

Obesity Associated With Defects in Leptin-melanocortin Pathway

Keywords

setmelanotide; RM-493; obesity; melanocortin 4 receptor; hypothalamic obesity

Outcome Measures

Primary Outcomes (1)

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

  Up to 3 years

Study Arms (1)

Setelanotide (Open-label)

EXPERIMENTAL

Once daily (QD) subcutaneous injection of setmelanotide

Drug: Setmelanotide

Interventions

Setmelanotide DRUG

Open-label daily injections of setmelanotide

Also known as: RM-493, Imcivree

Setelanotide (Open-label)

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Rhythm Pharmaceuticals, Inc.: lead

Study Sites (13)

HonorHealth Research Institute

Scottsdale, Arizona, 85258, United States

Location

UC San Diego- Rady Children's Hospital

San Diego, California, 92123, United States

Location

University of Florida at Gainesville

Gainesville, Florida, 32611, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

Children's Minnesota

Saint Paul, Minnesota, 55102, United States

Location

University of Buffalo

Getzville, New York, 14068, United States

Location

M3 Wake Research, Inc

Raleigh, North Carolina, 27612, United States

Location

Geisinger Health System

Danville, Pennsylvania, 17821, United States

Location

WR-ClinSearch

Chattanooga, Tennessee, 37421, United States

Location

Vanderbilt University School of Medicine

Nashville, Tennessee, 37232, United States

Location

Seattle Children's Research Institute

Seattle, Washington, 98105, United States

Location

Marshfield Clinical Research Institute

Marshfield, Wisconsin, 54449, United States

Location

MeSH Terms

Conditions

Obesity; Sexual Infantilism

Interventions

setmelanotide

Condition Hierarchy (Ancestors)

Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Gonadal Disorders; Endocrine System Diseases; Hypogonadism

Study Officials

• David Meeker, MD

  Rhythm Pharmaceuticals, Inc.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2024
• First Posted: September 19, 2024
• Study Start: October 21, 2024
• Primary Completion (Estimated): October 30, 2027
• Study Completion (Estimated): October 30, 2027
• Last Updated: September 4, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (13)