Monogenic Obesity
Conditions
Keywords
semaglutide, monogenic obesity
Brief summary
This observational study aims to assess the effect of once-weekly s.c. semaglutide 2.4 mg as an adjunct to a calorie-reduced diet and increased physical activity on weight loss, change in hunger, body composition, depression, and quality of life after 68 weeks of treatment in adolescents diagnosed with monogenic obesity in routine clinical care.
Detailed description
The primary objective of this prospective, non-interventional observational study is to evaluate the effect of once-weekly s.c. semaglutide 2.4 mg in routine clinical care as an adjunct to a calorie-reduced diet and increased physical activity on weight loss after 68 weeks of treatment in adolescents diagnosed with monogenic obesity. The secondary objectives of this prospective, non-interventional observation are to evaluate treatment compliance and to assess the influence of once-weekly s.c. semaglutide 2.4 mg in clinical practice on hunger score, body mass parameters, body composition, and depression score after 68 weeks of treatment in adolescents diagnosed with monogenic obesity. In addition, we will document the known parameters of safety and tolerability to determine safety and tolerability in clinical practice. The exploratory objective of this prospective, non-interventional observation is to assess user satisfaction by measuring change in subjective hunger score, quality of life, and perceptions and attitudes regarding treatment with semaglutide in adolescents diagnosed with monogenic obesity treated in routine clinical practice with once-weekly s.c. semaglutide 2.4 mg after 68 weeks of treatment.
Interventions
Treatment with semaglutide as prescribed in routine clinical practice according to summary of product characteristics. The use of semaglutide can be categorised into two groups during enrolment period: 1. new users, or 2. current users (e.g., those continuing semaglutide treatment prescribed in routine clinical practice).
Sponsors
Novo Nordisk A/S
Prof. Dr. Martin Wabitsch
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to 21 Years
Healthy volunteers
No
Inclusion criteria
1. Treatment with semaglutide as prescribed in routine clinical practice according to summary of product characteristics. 2. Informed consent of the patient, their parents, or legally acceptable representative (LAR) of participant and adolescent assent, as age-appropriate. 3. Age at time of signing informed consent: ≥12 to \<21 years. 4. BMI ≥95th percentile as defined on sex- and age-specific BMI growth charts (CDC.gov) 5. Body weight of \>60 kg. 6. Diagnosis of monogenic obesity by a Clinical Laboratory Improvement Amendments (CLIA)/ College of American Pathologists (CAP)/International Organisation for Standardization (ISO) 1518-certified laboratory using ACMG criteria as pathogenic (P), likely pathogenic (LP) and variant of uncertain significance (VUS).
Exclusion criteria
1. Participation in any interventional clinical trials at the time of enrolment. 2. Females of childbearing potential who are not using adequate contraceptive methods (as required by local law or practice) 3. Hypersensitivity to the active substance or to any of the excipients listed: * Disodium phosphate, dihydrate * Propylene glycol * Phenol * Hydrochloric acid (for pH adjustment) * Sodium hydroxide (for pH adjustment) * Water for injection 4. The safety and efficacy of Wegovy have not been investigated in patients: * treated with other products for weight management, * with type 1 diabetes, * with severe renal impairment (see section 4.2), * with severe hepatic impairment (see section 4.2), * with congestive heart failure New York Heart Association (NYHA) class IV. Use in these patients is not recommended
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of participants achieving ≥10% BMI reduction from baseline (week 0) to week 68. | 68 weeks | The study evaluates the effect of once-weekly subcutaneous (s.c.) semaglutide 2.4 mg administered as an adjunct to a calorie-reduced diet and increased physical activity in adolescents diagnosed with monogenic obesity. This measure focuses on weight loss effectiveness, with the goal of assessing the impact of the treatment on BMI reduction in a real-world clinical setting after 68 weeks of treatment. The study aims to provide insight into the potential of semaglutide in managing weight in this specific patient population. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Secondary Endpoints for Weight and Health Parameters | 68 weeks | The secondary endpoints of this study focus on changes in health outcomes over 68 weeks. These include body weight and BMI, measured as percentages or in kg and kg/m², with specific thresholds (e.g., 95th percentile) for age- and sex-specific BMI. Blood pressure (systolic and diastolic) is recorded in mmHg. Cholesterol levels (total, HDL, LDL, triglycerides) are assessed in mg/dL to evaluate cardiovascular risk. HbA1c (percentage), fasting glucose (mg/dL), and insulin (pmol/L) are monitored for metabolic health. ALT (IU/L) measures liver function. Changes in fat and lean mass are evaluated by DXA (kg). Weight and BMI velocity are measured in percentage points. The proportion of participants achieving ≥5% and ≥15% BMI reduction is also reported, offering insight into the treatment's effectiveness on weight and metabolic health. |
Countries
France, Germany, Netherlands, Spain, United Kingdom
Contacts
Primary ContactMartin Wabitsch, Prof. Dr.
Backup ContactStefanie Zorn, Dr.
Outcome results
None listed