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| Name | Class |
|---|---|
| Novo Nordisk A/S | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with biallelic variants in the LEPR, PCSK1, POMC, and MC4R gene |
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| Patients with monoallelic variants in the LEPR gene |
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| Patients with monoallelic variants in the PCSK1 gene |
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| Patients with monoallelic variants in the POMC gene |
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| Patients with monoallelic variants in the MC4R gene |
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| Patients with monoallelic variants in the SH2B1 gene or with 16p11.2 deletions |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Semaglutide (administered by PDS290 pen-injector) | Drug | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants achieving ≥10% BMI reduction from baseline (week 0) to week 68. | 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. | 68 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Secondary Endpoints for Weight and Health Parameters | 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. |
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Inclusion Criteria:
Exclusion Criteria:
Participation in any interventional clinical trials at the time of enrolment.
Females of childbearing potential who are not using adequate contraceptive methods (as required by local law or practice)
Hypersensitivity to the active substance or to any of the excipients listed:
The safety and efficacy of Wegovy have not been investigated in patients:
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Patients aged ≥12 to <21 years diagnosed with monogenic obesity, who agreed on treatment with semaglutide, are eligible for study participation.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Martin Wabitsch, Prof. Dr. | Contact | +4973150057401 | martin.wabitsch@uniklinik-ulm.de | |
| Stefanie Zorn, Dr. | Contact | +4973150057457 | stefanie.zorn@uniklinik-ulm.de |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Assistance Publique-Hôpitaux de Paris (AP-HP), Trousseau Hospital Paris, Pediatric Nutrition Department | Not yet recruiting | Paris | France |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34291582 | Background | Welling MS, de Groot CJ, Kleinendorst L, van der Voorn B, Burgerhart JS, van der Valk ES, van Haelst MM, van den Akker ELT, van Rossum EFC. Effects of glucagon-like peptide-1 analogue treatment in genetic obesity: A case series. Clin Obes. 2021 Dec;11(6):e12481. doi: 10.1111/cob.12481. Epub 2021 Jul 21. | |
| 33205056 | Background |
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Due to participant confidentiality requirements under GDPR regulations, IPD will not be shared. The study design also limits data sharing at this stage
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Serum, plasma, whole blood (EDTA), fat tissue
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| 68 weeks |
| Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin | Not yet recruiting | Berlin | 13353 | Germany |
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| University Hospital for Children and Adolescents, Center for Pediatric Research, Medical Faculty, University of Leipzig | Not yet recruiting | Leipzig | 04103 | Germany |
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| Division of Paediatric Endocrinology and Diabetes, Department of Paediatrics and Adolescent Medicine, Ulm University Medical Centre | Recruiting | Ulm | 89075 | Germany |
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| University Medical Center Rotterdam, Erasmus MC-Sophia Children's Hospital | Not yet recruiting | Rotterdam | 3015 GD | Netherlands |
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| Departments of Pediatrics & Pediatric Endocrinology, Hospital Infantil Universitario Niño Jesús, Universidad Autónoma de Madrid | Not yet recruiting | Madrid | 28009 | Spain |
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| Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre | Not yet recruiting | Cambridge | CB2 0QQ | United Kingdom |
|
| Iepsen EW, Have CT, Veedfald S, Madsbad S, Holst JJ, Grarup N, Pedersen O, Brandslund I, Holm JC, Hansen T, Torekov SS. GLP-1 Receptor Agonist Treatment in Morbid Obesity and Type 2 Diabetes Due to Pathogenic Homozygous Melanocortin-4 Receptor Mutation: A Case Report. Cell Rep Med. 2020 Apr 21;1(1):100006. doi: 10.1016/j.xcrm.2020.100006. eCollection 2020 Apr 21. |
| 35528826 | Background | Wabitsch M, Farooqi S, Fluck CE, Bratina N, Mallya UG, Stewart M, Garrison J, van den Akker E, Kuhnen P. Natural History of Obesity Due to POMC, PCSK1, and LEPR Deficiency and the Impact of Setmelanotide. J Endocr Soc. 2022 Apr 15;6(6):bvac057. doi: 10.1210/jendso/bvac057. eCollection 2022 Jun 1. |
| 34097736 | Background | Courbage S, Poitou C, Le Beyec-Le Bihan J, Karsenty A, Lemale J, Pelloux V, Lacorte JM, Carel JC, Lecomte N, Storey C, De Filippo G, Coupaye M, Oppert JM, Tounian P, Clement K, Dubern B. Implication of Heterozygous Variants in Genes of the Leptin-Melanocortin Pathway in Severe Obesity. J Clin Endocrinol Metab. 2021 Sep 27;106(10):2991-3006. doi: 10.1210/clinem/dgab404. |
| 28377240 | Background | Nordang GBN, Busk OL, Tveten K, Hanevik HI, Fell AKM, Hjelmesaeth J, Holla OL, Hertel JK. Next-generation sequencing of the monogenic obesity genes LEP, LEPR, MC4R, PCSK1 and POMC in a Norwegian cohort of patients with morbid obesity and normal weight controls. Mol Genet Metab. 2017 May;121(1):51-56. doi: 10.1016/j.ymgme.2017.03.007. Epub 2017 Mar 29. |
| ID | Term |
|---|---|
| C000591245 | semaglutide |
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