To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with LEPR deficiency obesity due to rare bi-allelic or loss-of function mutations at the end of 1 year of treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Once daily subcutaneous injection
Once daily subcutaneous injection
Markham Stouffville Hospital
Markham, Ontario, Canada
Institute of Cardiometabolism and Nutrition / Pitié-Salpêtrière Hospital
Paris, France
University of Ulm
Ulm, Germany
Erasmus Medical Center
Rotterdam, Netherlands
Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal Cohort)
The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\~1 year) of treatment were analyzed.
Time frame: Week 52
Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal + Supplemental Cohort)
The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\~1 year) of treatment were analyzed.
Time frame: Week 52
Mean Percent Change From Baseline in Body Weight
The mean percent change from baseline in body weight at 52 weeks was analyzed.
Time frame: Baseline and Week 52
Mean Percent Change From Baseline in Hunger Score ('Most Hunger')
The mean percent change in hunger scores for participants ≥12 years of age with leptin receptor (LEPR) deficiency obesity in treatment with setmelanotide was evaluated. Hunger score ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis.
Time frame: Baseline and Week 52
Percentage of Participants Achieving at Least 25% Improvement in Daily Hunger From Baseline
The percentage of participants (≥12 years of age) achieving a ≥25% improvement from baseline in hunger score at Week 52 (i.e., after treatment with setmelanotide for 52 weeks at the therapeutic dose) was assessed. Hunger ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis.
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Hôpital Félix GUYON
Saint-Denis, Reunion
University of Cambridge Metabolic Research Laboratories
Cambridge, United Kingdom
Time frame: Baseline and Week 52
Absolute Change From Baseline in Waist Circumference
Waist circumference (cm) was measured according to the National Heart, Lung, and Blood Institute (NHLBI) criteria. Waist circumference was measured when participants were fasting at approximately the same time at each visit. The absolute change from baseline in waist circumference was assessed.
Time frame: Baseline and Week 52
Absolute Change in Body Weight (Reversal of Weight Loss) During Double-Blind Placebo-Controlled Withdrawal Period
A comparison of weight change was evaluated during the 8 week placebo-controlled withdrawal period for each participant, during which each participant received 4 weeks of placebo and 4 weeks active therapy in a blinded fashion.
Time frame: Baseline and Week 8 of withdrawal period
Absolute Daily Hunger Reduction Score During the Double-Blind Placebo-Controlled Withdrawal Period
The absolute score in daily hunger reduction during the double-blind placebo-controlled withdrawal period (≥12 Years of Age) was assessed. Hunger ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis. The weekly average hunger score of the daily worst (most) hunger score in 24 hours is the hunger score used to assess this study endpoint. Lower scores represent lower hunger, higher scores represent greater hunger.
Time frame: Week 8 of withdrawal period
Mean Percent Change From Baseline in Body Mass Index
The mean percent change from baseline in body mass index (BMI) was assessed.
Time frame: Baseline and Week 52
Change From Baseline in Glucose Parameters
Glucose parameters included glucose, glycated hemoglobin (HbA1c) and Oral glucose tolerance test (OGTT). Data is planned to be reported only for change from baseline in glucose levels.
Time frame: Baseline and Week 52