Long Term Extension Trial of Setmelanotide

May 20, 2025 updated by: Rhythm Pharmaceuticals, Inc.

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
205

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2E1
        • University of Alberta
  • France
      • Paris, France, 75013
        • Hôpital de la Pitié Salpêtrière
      • Paris, France, 75012
        • Hôpital TROUSSEAU
  • Germany
      • Berlin, Germany, 13354
        • Charité - Universitätsmedizin Berlin
      • Leipzig, Germany, 04103
        • University of Leipzig
      • Ulm, Germany, 89075
        • University of Ulm
  • Greece
      • Rio, Greece, 26504
        • University General Hospital of Patras
  • Netherlands
      • Rotterdam, Netherlands
        • Erasmus MC
  • Spain
      • Madrid, Spain, 65 28009
        • Universidad Autonoma de Madrid
  • United Kingdom
      • Birmingham, United Kingdom, B15 2GW
        • University Hospitals Birmingham NHS Foundation Trust
      • Cambridge, United Kingdom, CB2 0QQ
        • Wellcome Trust-MRC Institute of Metabolic Science
      • London, United Kingdom, W12 0NN
        • Hammersmith Hospital
  • United States
    • Arizona
      • Chandler, Arizona, United States, 85224
        • Synexus Clinical Research US, Inc. - Phoenix Southeast
      • Scottsdale, Arizona, United States, 85258
        • Honor Health Research Institute
    • California
      • San Diego, California, United States, 92108
        • San Diego Wake Research
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida College of Medicine
    • Massachusetts
      • Springfield, Massachusetts, United States, 01107
        • Baystate Medical Center
    • New York
      • Buffalo, New York, United States, 14203
        • University at Buffalo
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center
      • Raleigh, North Carolina, United States, 27612
        • Wake Research
    • Pennsylvania
      • Danville, Pennsylvania, United States, 17822-2607
        • Obesity Institute, Geisinger Clinic
      • Philadelphia, Pennsylvania, United States, 19104
        • Childrens Hospital of Philadephia
    • South Carolina
      • Anderson, South Carolina, United States, 29621
        • Synexus Clinical Research US, Inc.- Primary Care Associates, PC
    • Tennessee
      • Chattanooga, Tennessee, United States, 37421
        • Wake Research TN
      • Nashville, Tennessee, United States, 37212
        • Vanderbilt University Medical Center
    • Washington
      • Seattle, Washington, United States, 98101
        • Seattle Children's Research Institute
    • Wisconsin
      • Marshfield, Wisconsin, United States, 54449
        • Marshfield Clinic Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: 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.
Drug: Setmelanotide
Once daily subcutaneous injection
Other Names:
  • RM-493

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
From first dose up to 5.6 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Rhythm Pharmaceuticals, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Chair: David Meeker, MD, Rhythm Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 15, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 9, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 9, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 18, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 27, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 29, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 31, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 20, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • RM-493-022
  • 2017-005006-35 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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