Long Term Extension Trial of Setmelanotide

Phase 2

Completed

• Conditions: Obesity Associated With Defects in Leptin-melanocortin Pathway
• Interventions: Drug: Setmelanotide

• Registration Number: NCT03651765
• Lead Sponsor: Rhythm Pharmaceuticals, Inc.

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.

Study Timeline

• Study First Submitted: 2018-01-18
• Study Start: 2018-07-15
• Study First Submitted QC: 2018-08-27
• Study First Posted: 2018-08-29
• Primary Completion: 2025-01-09
• Study Completion: 2025-01-09
• Status Verified: 2025-05
• Results First Submitted: 2025-05-20
• Results First Submitted QC: 2025-05-20
• Last Update Submitted: 2025-05-20
• Results First Posted: 2025-05-31
• Last Update Posted: 2025-05-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 205

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Setmelanotide	Setmelanotide	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	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.

Trial Locations

Locations (27)

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

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