Open-Label Extension Study of Setmelanotide

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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06596135

Enrollment

Registered

2024-09-19

Start date

2024-10-21

Completion date

2026-06-24

Last updated

2026-07-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Obesity Associated With Defects in Leptin-melanocortin Pathway

Keywords

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

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.

Interventions

DRUGSetmelanotide

Open-label daily injections of setmelanotide

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

Inclusion criteria

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/

Design outcomes

Primary

Measure	Time frame
Safety and tolerability of setmelanotide assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)	Up to 3 years

Countries

United States

Contacts

STUDY_CHAIRDavid Meeker, MD

Rhythm Pharmaceuticals, Inc.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 16, 2026