This is a sub-study of Study RM-493-040 (NCT05774756). The goal of this sub-study is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with congenital Hypothalamic Obesity (cHO). To determine how well setmelanotide works and how safe it is, patients with cHO will take a daily injection of either setmelanotide or placebo and complete trial assessments for up to 26 weeks on a therapeutic regimen.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
39
Solution for daily subcutaneous injection
Placebo matched to setmelanotide for daily subcutaneous injection
University of Alabama
Birmingham, Alabama, United States
RECRUITINGChildren's Hospital Colorado
Aurora, Colorado, United States
RECRUITINGLurie Children's Hospital
Chicago, Illinois, United States
RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
RECRUITINGChildren's Minnesota
Saint Paul, Minnesota, United States
RECRUITINGColumbia University Irving Medical Center
New York, New York, United States
RECRUITINGChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGSeattle Children's Research Institute
Seattle, Washington, United States
RECRUITINGUCL Great Ormond Street Institute of Child Health
London, Greater London, United Kingdom
RECRUITINGBirmingham Women and Children's Hospital NHS Trust
Birmingham, West Midlands, United Kingdom
RECRUITING...and 1 more locations
Mean % change in BMI
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with ≥5% reduction in BMI in adult patients (≥18 years of age) or a BMI Z-score reduction of ≥0.2 points in pediatric patients (<18 years of age)
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Mean change in the weekly average of the daily most hunger score in patients ≥12 years old
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with a ≥2 point reduction in the weekly average of the daily most hunger score
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Mean change in Symptoms of Hyperphagia total score
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with a ≥10% reduction in BMI
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Mean percent change in weight in patients ≥18 years
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Mean BMI-z score and BMI percentile reduction in patients <18 (using combined height and weight to report BMI in kg/m2)
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients aged ≥4 to <18 years with ≥0.2-point reduction of BMI Z-score
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Proportion of patients with BMI <30 kg/m2 (patients aged ≥18 years) or <95th percentile (patients aged <18 years)
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Mean change in physical functioning score and total score for the Impact of Weight on Quality of Life-Lite (IWQOL)
Time frame: From Baseline after 26 weeks on a therapeutic regimen
Safety and tolerability of setmelanotide compared to placebo assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Baseline after 26 weeks on a therapeutic regimen
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