Obesity Associated With Defects in Leptin-melanocortin PathwayPhase 3NCT06596135
Open-Label Extension Study of Setmelanotide
A Phase 3 study testing Setmelanotide in people with Obesity Associated With Defects in Leptin-melanocortin Pathway, run by Rhythm Pharmaceuticals, Inc. at 13 sites in United States.
Trial summary
This is an open-label extension study designed to evaluate the long-term safety and tolerability of continued setmelanotide treatment in male and female patients ≥2 years of age who have completed or transitioned from a previous study with setmelanotide for rare genetic, syndromic, or acquired diseases of obesity… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Obesity Associated With Defects in Leptin-melanocortin Pathway
- What is being tested?
- Setmelanotide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 13 sitesUnited States
- Study dates
- 21 Oct 2024to 24 Jun 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males and females ≥2 years of age who have completed a previous setmelanotide clinical study and who have a condition or indication for which Rhythm plans further clinical development.
- Agree to use a highly effective form of contraception and follow the study contraception requirements throughout study duration and for 90 days after.
- Demonstrated clinical benefit in the previous setmelanotide study, as determined by the Investigator.
Who cannot join
- Any gene variant, syndromic, or acquired disease for which setmelanotide is currently approved in the United States (i.e., patients eligible for commercial setmelanotide (IMCIVREE®).
- Any new or worsening depression resulting in suicidal thoughts and/or behaviors.
- Discontinuation from a previous setmelanotide study.
- History or close family history (parents or siblings) of melanoma.
- Pregnant and/or breastfeeding women
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
University of Michigan
Ann Arbor, Michigan, 48109
WR-ClinSearch
Chattanooga, Tennessee, 37421
Geisinger Health System
Danville, Pennsylvania, 17821
University of Florida at Gainesville
Gainesville, Florida, 32611
University of Buffalo
Getzville, New York, 14068
Marshfield Clinical Research Institute
Marshfield, Wisconsin, 54449
Vanderbilt University School of Medicine
Nashville, Tennessee, 37232
M3 Wake Research, Inc
Raleigh, North Carolina, 27612
Mayo Clinic - Rochester
Rochester, Minnesota, 55905
Children's Minnesota
Saint Paul, Minnesota, 55102
UC San Diego- Rady Children's Hospital
San Diego, California, 92123
HonorHealth Research Institute
Scottsdale, Arizona, 85258
Seattle Children's Research Institute
Seattle, Washington, 98105
About this study
Official title: Open-Label Extension Study of Setmelanotide in Patients With a Rare Genetic, Syndromic or Acquired Disease of the MC4R Pathway
This is an open-label extension study designed to evaluate the long-term safety and tolerability of continued setmelanotide treatment in male and female patients ≥2 years of age who have completed or transitioned from a previous study with setmelanotide for rare genetic, syndromic, or acquired diseases of obesity upstream of the MC4R pathway.
- Being tested
- Setmelanotide
- Keywords
- setmelanotide, RM-493, obesity, melanocortin 4 receptor, hypothalamic obesity
- Medical subject headings
- Obesity, Sexual Infantilism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SetmelanotideDrug
Also called RM-493, Imcivree
Open-label daily injections of setmelanotide
| Study group | Role | What they receive |
|---|---|---|
| Setelanotide (Open-label) | Experimental | Once daily (QD) subcutaneous injection of setmelanotide Receives: Drug: Setmelanotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of setmelanotide assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start21 Oct 2024
- Primary completion24 Jun 2026
- Study completion24 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Obesity Associated With Defects in Leptin-melanocortin PathwayStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Meeker, MD, Study chair, Rhythm Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06596135
- Sponsor's protocol ID
- RM-493-042
- Status last verified
- Jul 2026
- First submitted
- 10 Sep 2024
- Last updated
- 15 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Extension Study of Setmelanotide in Patients With a Rare Genetic, Syndromic or Acquired Disease of the MC4R Pathway
- ClinicalTrials.gov ID
- NCT06596135
- Acronym / sponsor ID
- RM-493-042
- Keywords
- setmelanotide, RM-493, obesity, melanocortin 4 receptor, hypothalamic obesity
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Oct 2024 (actual)
- Primary completion
- 24 Jun 2026 (actual)
- Study completion
- 24 Jun 2026 (actual)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label extension study designed to evaluate the long-term safety and tolerability of continued setmelanotide treatment in male and female patients ≥2 years of age who have completed or transitioned from a previous study with setmelanotide for rare genetic, syndromic, or acquired diseases of obesity upstream of the MC4R pathway.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Setelanotide (Open-label) | Experimental | Drug: SetmelanotideOnce daily (QD) subcutaneous injection of setmelanotide |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Males and females ≥2 years of age who have completed a previous setmelanotide clinical study and who have a condition or indication for which Rhythm plans further clinical development.
- Agree to use a highly effective form of contraception and follow the study contraception requirements throughout study duration and for 90 days after.
- Demonstrated clinical benefit in the previous setmelanotide study, as determined by the Investigator.
Key exclusion criteria (6 listed)
- Any gene variant, syndromic, or acquired disease for which setmelanotide is currently approved in the United States (i.e., patients eligible for commercial setmelanotide (IMCIVREE®).
- Any new or worsening depression resulting in suicidal thoughts and/or behaviors.
- Discontinuation from a previous setmelanotide study.
- History or close family history (parents or siblings) of melanoma.
- Pregnant and/or breastfeeding women
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SetmelanotideAlso: RM-493, ImcivreeOpen-label daily injections of setmelanotide | Drug | Setelanotide (Open-label) |
Primary outcome
Safety and tolerability of setmelanotide assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jul 2026.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Meeker, MD study chair
Rhythm Pharmaceuticals, Inc.
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Setmelanotide in people with Obesity Associated With Defects in Leptin-melanocortin Pathway, run by Rhythm Pharmaceuticals, Inc. at 13 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of setmelanotide assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Up to 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jul 2026.