Clinical trial NCT06772597: A Single-center Study of Setmelanotide in Patients With Prader-Willi Syndrome
Official title: A Phase 2, Single-center, Open-label Study of Setmelanotide in Patients With Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Prader-Willi Syndrome
Obesity
Hyperphagia
Keywords: melanocortin 4 receptor (MC4R), MC4R agonist
Lead sponsor: Rhythm Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2025-01-13
Enrollment: 20
Sex: ALL
Minimum age (years): 6
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility 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.

Primary outcomes:
measure: Frequency and severity of adverse events (AEs)
timeFrame: Baseline to Week 26
Secondary outcomes:
measure: Proportion of patients who achieve 5% reduction in BMI
timeFrame: Baseline to Week 26
measure: Mean percent change in BMI
timeFrame: Baseline to Week 26
measure: Mean percent change in BMI and weight in patients ≥18 years of age
timeFrame: Baseline to Week 26
measure: Mean change in BMI Z-Score and BMI % of 95th percentile
timeFrame: Baseline to Week 26
measure: 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
timeFrame: Baseline to Week 26
measure: Mean change in the weekly average of the Prader-Willi Syndrome Food Problem Diary (PWS-FPD) total score
timeFrame: Baseline to Week 26
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Florida
state: Florida
Study officials:
affiliation: Rhythm Pharmaceuticals, Inc.
name: David Meeker, MD
role: STUDY_CHAIR
References: