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

Trial Summary

Will I have to stop taking my current medications?

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

What is the purpose of this trial?

This is a Phase 2, single-center, 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 26 weeks.

Research Team

David Meeker, MD

Principal Investigator

Rhythm Pharmaceuticals, Inc.

Eligibility Criteria

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

I do not have severe heart, lung, metabolic, or cancer-related health issues that could affect the trial.

Timeline

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

Treatment Details

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.

Participant Groups

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+