Prader-Willi SyndromePhase 2NCT06772597
A Study of Setmelanotide in Patients With Prader-Willi Syndrome
A Phase 2 study testing Setmelanotide in people with Prader-Willi Syndrome and Obesity, run by Rhythm Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
This is a Phase 2, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age. Up to 20 patients are planned to be enrolled. Read more about this study
- Who is it for?
- Ages 6 to 65 years
- Conditions the study is for
- Prader-Willi Syndrome, Obesity, Hyperphagia
- 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
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Mar 2025to 31 Oct 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 6 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed diagnosis of Prader-Willi Syndrome (PWS)
- Age 6 to 65
- BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th Percentile for age and sex
- Agree to use a highly effective form of contraception and follow the study contraception requirements throughout study duration and for 90 days after.
- Patient and/or guardian is able to communicate well with the Investigator, understand and comply with the requirements of the study, and understand English and sign the written informed consent.
Who cannot join
- Use of weight modulating medications
- Abnormal eGFR, ALT, AST, and bilirubin values
- Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results.
- Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose
- Hypersensitivity to setmelanotide
- Diagnosis of severe psychiatric disorders
- Pregnant and/or breastfeeding
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Florida
Gainesville, Florida, 32610-0296
About this study
Official title: A Phase 2 Open-label Study of Setmelanotide in Patients With Prader-Willi Syndrome
This is a Phase 2, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age. Up to 20 patients are planned to be enrolled. Patients will take a daily subcutaneous dose of open-label setmelanotide for up to 52 weeks.
- Conditions
- Prader-Willi Syndrome, Obesity, Hyperphagia
- Being tested
- Setmelanotide
- Keywords
- melanocortin 4 receptor (MC4R), MC4R agonist
- Medical subject headings
- Prader-Willi Syndrome, Obesity, Hyperphagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SetmelanotideDrug
Also called Imcivree
Setmelanotide (daily subcutaneous injection)
| Study group | Role | What they receive |
|---|---|---|
| Setmelanotide (Open-label) | Experimental | Not described Receives: Drug: Setmelanotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of adverse events (AEs)
Measured: Baseline to Week 52
Secondary outcomes (6)
Proportion of patients who achieve 5% reduction in BMI
Measured: Baseline to Week 52
Mean percent change in BMI
Measured: Baseline to Week 52
Mean percent change in BMI and weight in patients ≥18 years of age
Measured: Baseline to Week 52
Mean change in BMI Z-Score and BMI % of 95th percentile
Measured: Baseline to Week 52
Proportion of patients who achieved 5% reduction in weight from Baseline in patients ≥18 years of age and who achieved ≥0.2 reduction in BMI Z-score in patients <18 years of age
Measured: Baseline to Week 52
Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score
Measured: Baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2025
- Study start4 Mar 2025
- Primary completion31 Oct 2026 (estimated)
- Study completion31 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 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
- NCT06772597
- Sponsor's protocol ID
- RM-493-043
- Status last verified
- Feb 2026
- First submitted
- 10 Jan 2025
- Last updated
- 12 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-label Study of Setmelanotide in Patients With Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT06772597
- Acronym / sponsor ID
- RM-493-043
- Conditions
- Prader-Willi Syndrome, Obesity, Hyperphagia
- Keywords
- melanocortin 4 receptor (MC4R), MC4R agonist
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Rhythm Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Mar 2025 (actual)
- Primary completion
- 31 Oct 2026 (estimated)
- Study completion
- 31 Oct 2027 (estimated)
- First posted
- 13 Jan 2025 (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 a Phase 2, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age. Up to 20 patients are planned to be enrolled. Patients will take a daily subcutaneous dose of open-label setmelanotide for up to 52 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Setmelanotide (Open-label) | Experimental | Drug: Setmelanotide |
Who may qualify
- Ages
- 6 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Confirmed diagnosis of Prader-Willi Syndrome (PWS)
- Age 6 to 65
- BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th Percentile for age and sex
- Agree to use a highly effective form of contraception and follow the study contraception requirements throughout study duration and for 90 days after.
- Patient and/or guardian is able to communicate well with the Investigator, understand and comply with the requirements of the study, and understand English and sign the written informed consent.
Key exclusion criteria (8 listed)
- Use of weight modulating medications
- Abnormal eGFR, ALT, AST, and bilirubin values
- Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered severe enough to interfere with the trial and/or confound the results.
- Participation in any clinical trial with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first trial dose
- Hypersensitivity to setmelanotide
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: ImcivreeSetmelanotide (daily subcutaneous injection) | Drug | Setmelanotide (Open-label) |
Primary outcome
Frequency and severity of adverse events (AEs)
Time frame: Baseline to Week 52
6 secondary outcomes
Proportion of patients who achieve 5% reduction in BMI
Time frame: Baseline to Week 52
Mean percent change in BMI
Time frame: Baseline to Week 52
Mean percent change in BMI and weight in patients ≥18 years of age
Time frame: Baseline to Week 52
Mean change in BMI Z-Score and BMI % of 95th percentile
Time frame: Baseline to Week 52
Proportion of patients who achieved 5% reduction in weight from Baseline in patients ≥18 years of age and who achieved ≥0.2 reduction in BMI Z-score in patients <18 years of age
Time frame: Baseline to Week 52
Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score
Time frame: Baseline to Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2026.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Setmelanotide in people with Prader-Willi Syndrome and Obesity, run by Rhythm Pharmaceuticals, Inc. at 1 site 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 5 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 12 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Frequency and severity of adverse events (AEs) (time frame: Baseline to Week 52). 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 12 Feb 2026.