Setmelanotide for Prader-Willi Syndrome

Overseen By Physician Inquiry Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Rhythm Pharmaceuticals, Inc.

Must not be taking: Weight modulating medications

Disqualifiers: Severe psychiatric disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called setmelanotide for individuals with Prader-Willi Syndrome (PWS), a condition often leading to obesity. The main goal is to assess the safety and effectiveness of daily setmelanotide use over a year. It targets those with a confirmed PWS diagnosis who struggle with obesity, specifically adults with a BMI of 30 or higher. Participants must communicate in English and agree to follow specific health guidelines during and after the trial. As a Phase 2 trial, this research measures setmelanotide's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in PWS treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking any weight modulating medications before participating.

Is there any evidence suggesting that setmelanotide is likely to be safe for humans?

Research has shown that setmelanotide is safe for treating obesity-related conditions. Studies have found that patients tolerate the drug well, with no new safety issues in recent tests. This includes data from past research where the treatment was used in everyday settings, confirming its long-term safety. While this trial focuses on obesity due to Prader-Willi Syndrome, the existing safety data for setmelanotide in similar conditions remains encouraging. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Prader-Willi Syndrome?

Setmelanotide is unique because it works by targeting the melanocortin-4 receptor (MC4R) pathway, which is crucial in regulating hunger and energy balance. Unlike other treatments for Prader-Willi Syndrome, which primarily manage symptoms through behavioral interventions and hormone treatments like growth hormone therapy, Setmelanotide directly addresses the root cause of excessive hunger associated with this condition. Researchers are excited about its potential to effectively reduce appetite and promote weight loss, offering a more direct and potentially more effective approach than current standard treatments.

What evidence suggests that setmelanotide might be an effective treatment for Prader-Willi Syndrome?

Studies have shown that setmelanotide can help people with obesity lose a significant amount of weight. In one study, participants lost 20% of their highest weight before treatment within the first year of using the drug. Early results from another trial with people who have Prader-Willi Syndrome (PWS) showed promising weight loss when the drug was taken daily for two months. These findings suggest that setmelanotide, which participants in this trial will receive, could help people with PWS manage their weight. This treatment targets specific parts of the body that control hunger and energy use. ^{1 2 3 4 6}

Who Is on the Research Team?

David Meeker, MD

Principal Investigator

Rhythm Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

This trial is for individuals aged 6 to 65 with Prader-Willi Syndrome who are significantly overweight. They must be able to follow the study's rules and agree to use effective birth control. People with severe psychiatric disorders, allergies to setmelanotide, serious health issues, recent participation in other drug trials, abnormal blood tests or those pregnant/breastfeeding cannot join.

Inclusion Criteria

I have been diagnosed with Prader-Willi Syndrome.

My BMI is 30 or higher, or in the top 95% for my 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

Exclusion Criteria

Diagnosis of severe psychiatric disorders

Hypersensitivity to setmelanotide

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a daily subcutaneous dose of open-label setmelanotide

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Setmelanotide

Trial Overview The trial is testing Setmelanotide's safety and effectiveness for reducing excessive eating and obesity in Prader-Willi Syndrome patients. Up to 20 participants will self-administer a daily injection of this medication for up to half a year.

How Is the Trial Designed?

Group I: Setmelanotide (Open-label) Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Rhythm Pharmaceuticals, Inc.

Lead Sponsor

Trials

31

Recruited

10,400+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40897637/

Real-World Efficacy and Safety of Setmelanotide in Adults ... Results: Patients experienced a clinically significant weight reduction of 20% from their highest pre-treatment weight within the first year.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06772597

NCT06772597 | A Study of Setmelanotide in Patients With ... Study Overview​​ 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 ...

3. praderwillinews.com praderwillinews.com/news/first-trial-data-setmelanotide-pws-obesity-expected-2025/

First trial data on setmelanotide for PWS obesity expected ... In a previous Rhythm-sponsored Phase 2 trial (NCT02311673), high doses of setmelanotide, taken once daily for two months, led to a reduction in ...

4. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/565716

A 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) ...

5. ir.rhythmtx.com ir.rhythmtx.com/node/11851/pdf

Rhythm Pharmaceuticals Announces Preliminary Fourth ... The primary endpoint for each substudy is the difference in mean percent change in BMI from baseline to 52 weeks in setmelanotide arm compared ...

6. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/abs/10.1002/oby.70002

Real‐World Efficacy and Safety of Setmelanotide in Adults ... The overall safety profile was consistent with phase 3 trials data, without any new safety signals. Conclusions. These findings confirm the ...