Clinical trial NCT06596135: Open-Label Extension Study of Setmelanotide
Official title: Open-Label Extension Study of Setmelanotide in Patients With a Rare Genetic, Syndromic or Acquired Disease of the MC4R Pathway
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Obesity Associated With Defects in Leptin-melanocortin Pathway
Keywords: setmelanotide
RM-493
obesity
melanocortin 4 receptor
hypothalamic obesity
Lead sponsor: Rhythm Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2024-09-19
Enrollment: 30
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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/Exclusion criteria may apply.

Primary outcomes:
measure: Safety and tolerability of setmelanotide assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Up to 3 years
Secondary outcomes:
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: HonorHealth Research Institute
state: Arizona
city: San Diego
country: United States
facility: UC San Diego- Rady Children's Hospital
state: California
city: Gainesville
country: United States
facility: University of Florida at Gainesville
state: Florida
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Saint Paul
country: United States
facility: Children's Minnesota
state: Minnesota
city: Getzville
country: United States
facility: University of Buffalo
state: New York
city: Raleigh
country: United States
facility: M3 Wake Research, Inc
state: North Carolina
city: Danville
country: United States
facility: Geisinger Health System
state: Pennsylvania
city: Chattanooga
country: United States
facility: WR-ClinSearch
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University School of Medicine
state: Tennessee
city: Seattle
country: United States
facility: Seattle Children's Research Institute
state: Washington
city: Marshfield
country: United States
facility: Marshfield Clinical Research Institute
state: Wisconsin
Study officials:
affiliation: Rhythm Pharmaceuticals, Inc.
name: David Meeker, MD
role: STUDY_CHAIR
References: