Ignite Creation Date:
2025-12-25 @ 3:46 AM
Ignite Modification Date:
2025-12-26 @ 2:33 AM
Study NCT ID:
NCT07302802
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-24
First Post:
2025-12-10
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy of Semaglutide s.c. Once-weekly on Weight Loss and Management in Adolescents With Monogenic Obesity in Clinical Practice
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C000591245', 'term': 'semaglutide'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Serum, plasma, whole blood (EDTA), fat tissue'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 70}, 'targetDuration': '68 Weeks', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2025-12-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2028-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-10', 'studyFirstSubmitDate': '2025-12-10', 'studyFirstSubmitQcDate': '2025-12-10', 'lastUpdatePostDateStruct': {'date': '2025-12-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-12-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of participants achieving ≥10% BMI reduction from baseline (week 0) to week 68.', 'timeFrame': '68 weeks', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'Secondary Endpoints for Weight and Health Parameters', 'timeFrame': '68 weeks', 'description': "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."}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['semaglutide', 'monogenic obesity'], 'conditions': ['Monogenic Obesity']}, 'referencesModule': {'references': [{'pmid': '34291582', 'type': 'BACKGROUND', 'citation': '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.'}, {'pmid': '33205056', 'type': 'BACKGROUND', 'citation': '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.'}, {'pmid': '35528826', 'type': 'BACKGROUND', 'citation': '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.'}, {'pmid': '34097736', 'type': 'BACKGROUND', 'citation': '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.'}, {'pmid': '28377240', 'type': 'BACKGROUND', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.\n\nThe 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.\n\nThe 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'minimumAge': '12 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients aged ≥12 to \\<21 years diagnosed with monogenic obesity, who agreed on treatment with semaglutide, are eligible for study participation.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Treatment with semaglutide as prescribed in routine clinical practice according to summary of product characteristics.\n2. Informed consent of the patient, their parents, or legally acceptable representative (LAR) of participant and adolescent assent, as age-appropriate.\n3. Age at time of signing informed consent: ≥12 to \\<21 years.\n4. BMI ≥95th percentile as defined on sex- and age-specific BMI growth charts (CDC.gov)\n5. Body weight of \\>60 kg.\n6. 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).\n\nExclusion Criteria:\n\n1. Participation in any interventional clinical trials at the time of enrolment.\n2. Females of childbearing potential who are not using adequate contraceptive methods (as required by local law or practice)\n3. Hypersensitivity to the active substance or to any of the excipients listed:\n\n * Disodium phosphate, dihydrate\n * Propylene glycol\n * Phenol\n * Hydrochloric acid (for pH adjustment)\n * Sodium hydroxide (for pH adjustment)\n * Water for injection\n4. The safety and efficacy of Wegovy have not been investigated in patients:\n\n * treated with other products for weight management,\n * with type 1 diabetes,\n * with severe renal impairment (see section 4.2),\n * with severe hepatic impairment (see section 4.2),\n * with congestive heart failure New York Heart Association (NYHA) class IV. Use in these patients is not recommended'}, 'identificationModule': {'nctId': 'NCT07302802', 'briefTitle': 'Efficacy of Semaglutide s.c. Once-weekly on Weight Loss and Management in Adolescents With Monogenic Obesity in Clinical Practice', 'organization': {'class': 'OTHER', 'fullName': 'University of Ulm'}, 'officialTitle': 'Efficacy of Semaglutide s.c. Once-weekly on Weight Loss and Management in Adolescents With Monogenic Obesity in Clinical Practice', 'orgStudyIdInfo': {'id': 'U1111-1307-1203'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Patients with biallelic variants in the LEPR, PCSK1, POMC, and MC4R gene', 'interventionNames': ['Drug: Semaglutide (administered by PDS290 pen-injector)']}, {'label': 'Patients with monoallelic variants in the LEPR gene', 'interventionNames': ['Drug: Semaglutide (administered by PDS290 pen-injector)']}, {'label': 'Patients with monoallelic variants in the PCSK1 gene', 'interventionNames': ['Drug: Semaglutide (administered by PDS290 pen-injector)']}, {'label': 'Patients with monoallelic variants in the POMC gene', 'interventionNames': ['Drug: Semaglutide (administered by PDS290 pen-injector)']}, {'label': 'Patients with monoallelic variants in the MC4R gene', 'interventionNames': ['Drug: Semaglutide (administered by PDS290 pen-injector)']}, {'label': 'Patients with monoallelic variants in the SH2B1 gene or with 16p11.2 deletions', 'interventionNames': ['Drug: Semaglutide (administered by PDS290 pen-injector)']}], 'interventions': [{'name': 'Semaglutide (administered by PDS290 pen-injector)', 'type': 'DRUG', 'description': '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).', 'armGroupLabels': ['Patients with biallelic variants in the LEPR, PCSK1, POMC, and MC4R gene', 'Patients with monoallelic variants in the LEPR gene', 'Patients with monoallelic variants in the MC4R gene', 'Patients with monoallelic variants in the PCSK1 gene', 'Patients with monoallelic variants in the POMC gene', 'Patients with monoallelic variants in the SH2B1 gene or with 16p11.2 deletions']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Paris', 'country': 'France', 'contacts': [{'name': 'Béatrice Dubern, Prof. Dr.', 'role': 'CONTACT', 'email': 'beatrice.dubern@aphp.fr'}], 'facility': 'Assistance Publique-Hôpitaux de Paris (AP-HP), Trousseau Hospital Paris, Pediatric Nutrition Department', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '13353', 'city': 'Berlin', 'country': 'Germany', 'contacts': [{'name': 'Peter Kühnen, Prof. Dr.', 'role': 'CONTACT', 'email': 'peter.kuehnen@charite.de'}], 'facility': 'Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '04103', 'city': 'Leipzig', 'country': 'Germany', 'contacts': [{'name': 'Antje Körner, Prof. Dr.', 'role': 'CONTACT', 'email': 'antje.koerner@medizin.uni-leipzig.de'}], 'facility': 'University Hospital for Children and Adolescents, Center for Pediatric Research, Medical Faculty, University of Leipzig', 'geoPoint': {'lat': 51.33962, 'lon': 12.37129}}, {'zip': '89075', 'city': 'Ulm', 'country': 'Germany', 'contacts': [{'name': 'Martin Wabitsch, Prof. Dr.', 'role': 'CONTACT', 'email': 'martin.wabitsch@uniklinik-ulm.de', 'phone': '+4973150057401'}, {'name': 'Stefanie Zorn, Dr.', 'role': 'CONTACT', 'email': 'stefanie.zorn@uniklinik-ulm.de', 'phone': '+4973150057457'}], 'facility': 'Division of Paediatric Endocrinology and Diabetes, Department of Paediatrics and Adolescent Medicine, Ulm University Medical Centre', 'geoPoint': {'lat': 48.39841, 'lon': 9.99155}}, {'zip': '3015 GD', 'city': 'Rotterdam', 'country': 'Netherlands', 'contacts': [{'name': 'Erica van den Akker, Prof. Dr.', 'role': 'CONTACT', 'email': 'e.l.t.vandenakker@erasmusmc.nl'}], 'facility': "University Medical Center Rotterdam, Erasmus MC-Sophia Children's Hospital", 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'zip': '28009', 'city': 'Madrid', 'country': 'Spain', 'contacts': [{'name': 'Jesús Argente, Prof. Dr.', 'role': 'CONTACT', 'email': 'jesus.argente@fundacionendo.org'}], 'facility': 'Departments of Pediatrics & Pediatric Endocrinology, Hospital Infantil Universitario Niño Jesús, Universidad Autónoma de Madrid', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': 'CB2 0QQ', 'city': 'Cambridge', 'country': 'United Kingdom', 'contacts': [{'name': 'Sadaf Farooqi, Prof. Dr.', 'role': 'CONTACT', 'email': 'isf20@medschl.cam.ac.uk'}], 'facility': 'Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}], 'centralContacts': [{'name': 'Martin Wabitsch, Prof. Dr.', 'role': 'CONTACT', 'email': 'martin.wabitsch@uniklinik-ulm.de', 'phone': '+4973150057401'}, {'name': 'Stefanie Zorn, Dr.', 'role': 'CONTACT', 'email': 'stefanie.zorn@uniklinik-ulm.de', 'phone': '+4973150057457'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'Due to participant confidentiality requirements under GDPR regulations, IPD will not be shared. The study design also limits data sharing at this stage'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Prof. Dr. Martin Wabitsch', 'class': 'OTHER'}, 'collaborators': [{'name': 'Novo Nordisk A/S', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'Prof. Dr. Martin Wabitsch', 'investigatorAffiliation': 'University of Ulm'}}}}