Clinical trial NCT03651765: Long Term Extension Trial of Setmelanotide
Official title: Long Term Extension Trial of Setmelanotide (RM-493) for Patients Who Have Completed a Trial of Setmelanotide for the Treatment of Obesity Associated With Genetic Defects Upstream of the MC4 Receptor in the Leptin-melanocortin Pathway
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Obesity Associated With Defects in Leptin-melanocortin Pathway
Keywords: setmelanotide
RM-493
obesity
leptin-melanocortin
melanocortin 4 receptor
Lead sponsor: Rhythm Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2018-08-29
Enrollment: 205
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label treatment
Primary purpose: TREATMENT

Brief summary: This was a long-term extension trial to study the safety and tolerability of continued setmelanotide treatment in participants who had completed a previous clinical trial on treatment with setmelanotide for obesity associated with genetic defects upstream of the Melanocortin-4 (MC4) receptor in the leptin-melanocortin pathway.

Detailed description: The primary objectives of this extension trial were to explore the long-term safety and tolerability of setmelanotide for up to 7 years or until drug was otherwise available through authorized use. Participants entered this protocol immediately upon completion of their index protocol such that dosing of setmelanotide continued without gaps in therapy.

Eligibility criteria: Key Inclusion Criteria:

1. Participants aged 2 or older (or aged \>2 years as per local regulations) who had completed participation in a previous setmelanotide trial and demonstrated adequate safety and meaningful clinical benefit (efficacy)
2. Participant and/or parent or guardian was able to communicate with the investigator, understand and sign the written informed consent/assent, and comply with the trial requirements
3. Agree to use a highly effective form of contraception throughout the trial

Key Exclusion Criteria:

1. Pregnant and/or breastfeeding women
2. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
3. Current, clinically significant disease
4. Documented diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s)
5. Suicidal ideation, attempt or behavior
6. History of significant liver disease
7. Moderate to severe renal dysfunction as defined by a glomerular filtration rate (GFR)\<30 milliliters per minute (mL/min).
8. History or close family history of melanoma or participant history of oculocutaneous albinism

Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
timeFrame: From first dose up to 5.6 years
Secondary outcomes:
Other outcomes:
Locations:
city: Chandler
country: United States
facility: Synexus Clinical Research US, Inc. - Phoenix Southeast
state: Arizona
city: Scottsdale
country: United States
facility: Honor Health Research Institute
state: Arizona
city: San Diego
country: United States
facility: San Diego Wake Research
state: California
city: Gainesville
country: United States
facility: University of Florida College of Medicine
state: Florida
city: Springfield
country: United States
facility: Baystate Medical Center
state: Massachusetts
city: Buffalo
country: United States
facility: University at Buffalo
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Raleigh
country: United States
facility: Wake Research
state: North Carolina
city: Danville
country: United States
facility: Obesity Institute, Geisinger Clinic
state: Pennsylvania
city: Philadelphia
country: United States
facility: Childrens Hospital of Philadephia
state: Pennsylvania
city: Anderson
country: United States
facility: Synexus Clinical Research US, Inc.- Primary Care Associates, PC
state: South Carolina
city: Chattanooga
country: United States
facility: Wake Research TN
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Seattle
country: United States
facility: Seattle Children's Research Institute
state: Washington
city: Marshfield
country: United States
facility: Marshfield Clinic Research Institute
state: Wisconsin
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Paris
country: France
facility: Hôpital Trousseau
city: Paris
country: France
facility: Hôpital de la Pitié Salpêtrière
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Leipzig
country: Germany
facility: University of Leipzig
city: Ulm
country: Germany
facility: University of Ulm
city: Rio
country: Greece
facility: University General Hospital of Patras
city: Rotterdam
country: Netherlands
facility: Erasmus MC
city: Madrid
country: Spain
facility: Universidad Autónoma de Madrid
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust
city: Cambridge
country: United Kingdom
facility: Wellcome Trust-MRC Institute of Metabolic Science
city: London
country: United Kingdom
facility: Hammersmith Hospital
Study officials:
affiliation: Rhythm Pharmaceuticals, Inc.
name: David Meeker, MD
role: STUDY_CHAIR
References:
citation: Meyer JR, Krentz AD, Berg RL, Richardson JG, Pomeroy J, Hebbring SJ, Haws RM. Kidney failure in Bardet-Biedl syndrome. Clin Genet. 2022 Apr;101(4):429-441. doi: 10.1111/cge.14119.