Hyperphagia, Obesity, Prader-Willi Syndrome
Conditions
Keywords
melanocortin 4 receptor (MC4R), MC4R agonist
Brief 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. Patients will take a daily subcutaneous dose of open-label setmelanotide for up to 52 weeks.
Interventions
DRUGSetmelanotide
Setmelanotide (daily subcutaneous injection)
Sponsors
Rhythm Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 65 Years
Healthy volunteers
No
Inclusion 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 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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Frequency and severity of adverse events (AEs) | Baseline to Week 52 |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of patients who achieve 5% reduction in BMI | Baseline to Week 52 |
| Mean percent change in BMI | Baseline to Week 52 |
| Mean percent change in BMI and weight in patients ≥18 years of age | Baseline to Week 52 |
| Mean change in BMI Z-Score and BMI % of 95th percentile | 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 | Baseline to Week 52 |
| Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score | Baseline to Week 52 |
Countries
United States
Contacts
STUDY_CHAIRDavid Meeker, MD
Rhythm Pharmaceuticals, Inc.
Outcome results
None listed