DRUG
Setmelanotide
Once daily subcutaneous injection
Long Term Extension Trial of Setmelanotide
Status
Completed
Phase
Phase 2 / Phase 3
Enrollment
205
Locations
27
Results
Posted
Publications
2
Study summary
What the protocol is testing.
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.
Full 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.
Interventions
Treatment arms and agents.
Timeline
From registration to results.
First posted
Aug 29, 2018
Study start
Jul 15, 2018
Primary completion
Jan 9, 2025
Study completion
Jan 9, 2025
Results posted
May 31, 2025
Registry updated
May 31, 2025
Outcomes
What the study measures.
Primary outcomes
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame · From first dose up to 5.6 years
An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. A TEAE was defined as any AE that began or worsened in intensity on or after the date of the first administration of study drug.
Secondary outcomes
Not reported in the indexed record.
Eligibility
Who can take part.
- Minimum age
- 2 Years
- Maximum age
- Not reported
- Sex
- ALL
- Healthy volunteers
- No
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.
Study locations
27 registered sites.
Canada · France · Germany · Greece · Netherlands · Spain · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
Synexus Clinical Research US, Inc. - Phoenix Southeast
Chandler, Arizona, United States
Honor Health Research Institute
Scottsdale, Arizona, United States
San Diego Wake Research
San Diego, California, United States
University of Florida College of Medicine
Gainesville, Florida, United States
Baystate Medical Center
Springfield, Massachusetts, United States
University at Buffalo
Buffalo, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Wake Research
Raleigh, North Carolina, United States
Obesity Institute, Geisinger Clinic
Danville, Pennsylvania, United States
Childrens Hospital of Philadephia
Philadelphia, Pennsylvania, United States
Synexus Clinical Research US, Inc.- Primary Care Associates, PC
Anderson, South Carolina, United States
Wake Research TN
Chattanooga, Tennessee, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Seattle Children's Research Institute
Seattle, Washington, United States
Marshfield Clinic Research Institute
Marshfield, Wisconsin, United States
University of Alberta
Edmonton, Alberta, Canada
Hôpital Trousseau
Paris, France
Hôpital de la Pitié Salpêtrière
Paris, France
Charité - Universitätsmedizin Berlin
Berlin, Germany
University of Leipzig
Leipzig, Germany
University of Ulm
Ulm, Germany
University General Hospital of Patras
Rio, Greece
Erasmus MC
Rotterdam, Netherlands
Universidad Autónoma de Madrid
Madrid, Spain
Publications
Results and literature.
PMID 38697184 ↗Roth CL, Scimia C, Shoemaker AH, Gottschalk M, Miller J, Yuan G, Malhotra S, Abuzzahab MJ. Setmelanotide for the treatment of acquired hypothalamic obesity: a phase 2, open-label, multicentre trial. Lancet Diabetes Endocrinol. 2024 Jun;12(6):380-389. doi: 10.1016/S2213-8587(24)00087-1. Epub 2024 Apr 30.PMID 35112343 ↗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.
Related trials
More studies on Setmelanotide.
NCT IDStudyPhase / statusEnrollmentUpdated
Setmelanotide to Treat Obesity in a Patient With Pseudohypoparathyroidism Type 1a (PHP1a)
Massachusetts General Hospital · Pseudohypoparathyroidism Type 1a · Obesity
Phase 2
Enrolling by invitation
1
2026-07
Open-Label Extension Study of Setmelanotide
Rhythm Pharmaceuticals, Inc. · Obesity Associated With Defects in Leptin-melanocortin Pathway
Phase 3
Completed
28
2026-07
Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity
Tom Hühne · Bardet Biedl Syndrome (BBS) · Bardet Biedl Syndrome
Phase 4
Recruiting
200
2026-06
A Trial of Setmelanotide in Acquired Hypothalamic Obesity
Rhythm Pharmaceuticals, Inc. · Hypothalamic Obesity
Phase 3
Active, not recruiting
143
2026-05
A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
Rhythm Pharmaceuticals, Inc. · Hypothalamic Obesity · Multiple Pituitary Hormone Deficiency Genetic Form
Phase 3
Recruiting
39
2026-02
A Study of Setmelanotide in Patients With Prader-Willi Syndrome
Rhythm Pharmaceuticals, Inc. · Prader-Willi Syndrome · Obesity
Phase 2
Active, not recruiting
18
2026-02
EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway
Rhythm Pharmaceuticals, Inc. · Obesity · Genetic Obesity
Phase 3
Active, not recruiting
296
2026-02
DAYBREAK: A Study of Setmelanotide in Participants With Specific Gene Variants in the Melanocortin-4 Receptor (MC4R) Pathway
Rhythm Pharmaceuticals, Inc. · Genetic Obesity
Phase 2
Completed
164
2025-07
Related PeptideStat pages
Put the record in context.
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