Long Term Extension Trial of Setmelanotide

NCT03651765 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: RM-493-0222017-005006-35
• Study Type: interventional
• Target: 205
• Locations: 8 countries
• Sites: 27

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.

Conditions

Obesity Associated With Defects in Leptin-melanocortin Pathway

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

  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.

  From first dose up to 5.6 years

Study Arms (1)

Setmelanotide

EXPERIMENTAL

Participants received setmelanotide at the same starting dose as received in the index trials as a Subcutaneous (SC) injection once daily for up to 5.6 years in this extension trial.

Drug: Setmelanotide

Interventions

Setmelanotide DRUG

Once daily subcutaneous injection

Also known as: RM-493

Setmelanotide

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:

• 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)
• 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
• Agree to use a highly effective form of contraception throughout the trial

You may not qualify if:

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

Sponsors & Collaborators

• Rhythm Pharmaceuticals, Inc.: lead

Study Sites (27)

Synexus Clinical Research US, Inc. - Phoenix Southeast

Chandler, Arizona, 85224, United States

Location

Honor Health Research Institute

Scottsdale, Arizona, 85258, United States

Location

San Diego Wake Research

San Diego, California, 92108, United States

Location

University of Florida College of Medicine

Gainesville, Florida, 32610, United States

Location

Baystate Medical Center

Springfield, Massachusetts, 01107, United States

Location

University at Buffalo

Buffalo, New York, 14203, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Wake Research

Raleigh, North Carolina, 27612, United States

Location

Obesity Institute, Geisinger Clinic

Danville, Pennsylvania, 17822-2607, United States

Location

Childrens Hospital of Philadephia

Philadelphia, Pennsylvania, 19104, United States

Location

Synexus Clinical Research US, Inc.- Primary Care Associates, PC

Anderson, South Carolina, 29621, United States

Location

Wake Research TN

Chattanooga, Tennessee, 37421, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location

Seattle Children's Research Institute

Seattle, Washington, 98101, United States

Location

Marshfield Clinic Research Institute

Marshfield, Wisconsin, 54449, United States

Location

University of Alberta

Edmonton, Alberta, T6G 2E1, Canada

Location

Hôpital Trousseau

Paris, 75012, France

Location

Hôpital de la Pitié Salpêtrière

Paris, 75013, France

Location

Charité - Universitätsmedizin Berlin

Berlin, 13354, Germany

Location

University of Leipzig

Leipzig, 04103, Germany

Location

University of Ulm

Ulm, 89075, Germany

Location

University General Hospital of Patras

Rio, 26504, Greece

Location

Erasmus MC

Rotterdam, Netherlands

Location

Universidad Autónoma de Madrid

Madrid, 65 28009, Spain

Location

University Hospitals Birmingham NHS Foundation Trust

Birmingham, B15 2GW, United Kingdom

Location

Wellcome Trust-MRC Institute of Metabolic Science

Cambridge, CB2 0QQ, United Kingdom

Location

Hammersmith Hospital

London, W12 0NN, United Kingdom

Location

Related Publications (2)

• 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: 38697184 DERIVED

• 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.

  PMID: 35112343 DERIVED

MeSH Terms

Conditions

Obesity

Interventions

setmelanotide

Condition Hierarchy (Ancestors)

Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Rhythm Clinical Trials
• Organization: Rhythm Pharmaceuticals, Inc.

clinicaltrials@rhythmtx.com

857-264-4280

Study Officials

• David Meeker, MD

  Rhythm Pharmaceuticals, Inc.

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open label treatment

Study Record Dates

• First Submitted: January 18, 2018
• First Posted: August 29, 2018
• Study Start: July 15, 2018
• Primary Completion: January 9, 2025
• Study Completion: January 9, 2025
• Last Updated: May 31, 2025
• Results First Posted: May 31, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

DE (3); CA (1); GR (1); US (15); FR (2); ES (1); GB (3); NL (1)