A Single-center Study of Setmelanotide in Patients With Prader-Willi Syndrome

Rhythm Pharmaceuticals

Status and phase

Enrolling

Phase 2

Conditions

Prader-Willi Syndrome

Obesity

Hyperphagia

Treatments

Drug: Setmelanotide

Study type

Interventional

Funder types

Industry

Identifiers

NCT06772597

RM-493-043

Details and patient eligibility

About

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.

Enrollment

20 estimated patients

Sex

All

Ages

6 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

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.

Exclusion Criteria:

Use of weight modulating medications
Abnormal HbA1c, eGFR, ALT, AST, bilirubin, T4 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.