Phase 2 N=40 Randomized Quadruple-blind Treatment

Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome

Prader-Willi Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT02311673 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Jul 2023

Primary outcome: Primary: Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 — 4; 11; 6; 11 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Setmelanotide (Drug); Placebo (Drug)
Age: Pediatric, Adult, Older Adult · 16+ yrs
Sex: All
Sponsor: Rhythm Pharmaceuticals, Inc.
Primary completion: Oct 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2	4; 11; 6; 11; 0; 0	—
PRIMARY Number of Participants Who Experienced a TEAE - Period 3	3; 7; 4; 11; 0; 0	—
PRIMARY Number of Participants Who Experienced a TEAE - Period 4	17; 12; 0; 0; 0; 0	—
PRIMARY Mean Body Weight - Period 2	94.3; 92.7; 101.6; 102.2	—
PRIMARY Percent Change From Baseline in Body Weight - Period 2	-0.7; -0.8; 0.4; -0.4	0.420
PRIMARY Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2	31.0; 21.2; 20.2; 18.7	—
PRIMARY Percent Change From Baseline in Overall Score of Prader-Willi Syndrome (PWS) Hyperphagia Questionnaire - Period 2	20.2; -5.2; -5.0; -1.3	0.901
SECONDARY Percent Change From Baseline in Hyperphagic Drive Score of PWS Hyperphagia Questionnaire - Period 2	27.2; -3.6; -6.8; 6.6	0.840
SECONDARY Percent Change From Baseline in Hyperphagic Behaviors Score of PWS Hyperphagia Questionnaire - Period 2	19.1; -1.7; -3.0; -0.4	0.812
SECONDARY Percent Change From Baseline in Hyperphagic Severity Score of PWS Hyperphagia Questionnaire - Period 2	33.2; -0.7; 5.5; -16.7	0.988
SECONDARY Percent Change From Baseline in Overall Score of PWS Hyperphagia Questionnaire - Period 3	35.0; -1.8; 3.4; 2.3	0.943
SECONDARY Percent Change From Baseline in Overall Score of PWS Hyperphagia Questionnaire - Period 4	-0.9; -0.4	—
SECONDARY Mean Setmelanotide Trough Concentrations	0.790; 2.59; 5.63	—
SECONDARY Maximum Drug Concentration (Cmax) of Setmelanotide During a 24-Hour Steady-State Interval	39.1	—
SECONDARY Time to the Maximum Drug Concentration (Tmax) of Setmelanotide During a 24-Hour Steady-State Interval	6.00	—
SECONDARY Area Under the Drug Concentration-Time Curve From Time-Zero to 24 Hours Postdose (AUC24h) of Setmelanotide During a 24-Hour Steady-State Interval	569.4	—
SECONDARY Volume of Distribution (Vd) of Setmelanotide During a 24-Hour Steady-State Interval	52.8	—
SECONDARY Total Clearance (CL) of Setmelanotide During a 24-Hour Steady-State Interval	4.77	—
SECONDARY Change From Baseline in Body Weight - Period 2	-0.6; -0.8; 0.3; -0.4	0.439
SECONDARY Percent Change From Baseline in Body Weight - Period 3	1.3; 0.0; 0.0; 0.2	0.883
SECONDARY Percent Change From Baseline in Body Weight - Period 4	-0.0; 0.0	—
SECONDARY Percent Change From Baseline in Body Weight for Continuous Active and Continuous Placebo Treatments - Period 2 and 3	-0.7; -0.5; 0.7; -0.7; 0.2; -0.4	0.679
SECONDARY Percent Change From Baseline in Body Fat Measured Using Dual x-Ray Absorptiometry (DEXA) - Period 2	-0.6; -1.1; -0.7; -1.1	0.633
SECONDARY Number of Participants With Clinically Significant Percent Change From Baseline in Body Fat Measured Using DEXA - Period 4	0; 0	—
SECONDARY Percent Change From Baseline in Body Mass Measured Using DEXA - Period 2	-0.2; -1.1; -0.3; -0.9	0.712
SECONDARY Number of Participants With Clinically Significant Percent Change From Baseline in Body Mass Measured Using DEXA - Period 4	0; 0	—

Summary

The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related behavior. The study drug (setmelanotide and placebo) was administered in a blinded fashion.

Eligibility Criteria

Inclusion Criteria

PWS due to chromosome 15 micro-deletion, maternal uniparental disomy, or imprinting defect, confirmed by fluorescent in situ hybridization, chromosomal microarray, and/or methylation studies. Obese male or female participants weighing at least 50 kilograms (kg) with body mass index (BMI) ≥ 27 kilogram per square meter (kg/m²)
Age 16-65 years
If a participant has diagnosis of type 2 diabetes, following criteria must be met:
hemoglobin A1C (HbA1c) 150/90 mm Hg.
Liver disease or liver injury as indicated by abnormal liver function tests, aspartate aminotransferase, alkaline phosphatase, or serum bilirubin (> 1.5 x upper limit of normal for any of these tests) or history of hepatic cirrhosis.
History or presence of impaired renal function as indicated by clinically significantly abnormal creatinine, blood urea nitrogen, or urinary constituents (e.g., albuminuria) or moderate to severe renal dysfunction as defined by the Cockcroft-Gault equation ( 500 milliliters (mL) within 3 months.
Inadequate venous access.
History of low blood counts or recurring infections.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02311673). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.