Ignite Creation Date:
2025-12-24 @ 11:41 PM
Ignite Modification Date:
2025-12-28 @ 2:45 AM
Study NCT ID:
NCT04484051
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-02-16
First Post:
2020-07-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Growth Hormone Study in Adults With Prader-Willi Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Australia']}, 'conditionBrowseModule': {'meshes': [{'id': 'D011218', 'term': 'Prader-Willi Syndrome'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}], 'ancestors': [{'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D000096803', 'term': 'Imprinting Disorders'}, {'id': 'D009765', 'term': 'Obesity'}, {'id': 'D050177', 'term': 'Overweight'}, {'id': 'D044343', 'term': 'Overnutrition'}, {'id': 'D009748', 'term': 'Nutrition Disorders'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 25}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2023-03-24', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-02', 'completionDateStruct': {'date': '2026-10-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2023-02-15', 'studyFirstSubmitDate': '2020-07-15', 'studyFirstSubmitQcDate': '2020-07-21', 'lastUpdatePostDateStruct': {'date': '2023-02-16', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-07-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-09-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change in weight and waist-hip ratio', 'timeFrame': '36 months', 'description': 'Change in weight (in kg) and waist-hip ratio'}, {'measure': 'Change in blood pressure', 'timeFrame': '36 months', 'description': 'Change in blood pressure (in mmHg)'}, {'measure': 'Occurence of side-effects', 'timeFrame': '36 months', 'description': 'Occurrence of side-effects'}], 'primaryOutcomes': [{'measure': 'Change in lean body mass', 'timeFrame': '36 months', 'description': 'Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan'}], 'secondaryOutcomes': [{'measure': 'Change in fat mass', 'timeFrame': '36 months', 'description': 'Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan'}, {'measure': 'Change in bone density', 'timeFrame': '36 months', 'description': 'Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan'}, {'measure': 'Change in physical strength', 'timeFrame': '36 months', 'description': 'Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests'}, {'measure': 'Change in laboratory measurements', 'timeFrame': '36 months', 'description': 'Changes in the following laboratory measurements:\n\n* Glycosylated hemoglobin (mmol/mol)\n* Total cholesterol (mmol/L)\n* Low-density lipoprotein cholesterol (mmol/L)\n* High-density lipoprotein cholesterol (mmol/L)\n* Insulin-like growth factor 1 (nmol/L)\n* Free thyroxine 4 (pmol/L)\n* Luteinizing hormone (U/I)\n* Follicle stimulating hormone (U/I)\n* Estradiol or testosterone (nmol/L)\n* Sex hormone binding globulin (nmol/L)\n* Aspartate transaminase (U/L)\n* Alanine transaminase (U/L)\n* Alkaline phosphatase (U/L)\n* Gamma glutamyl transpeptidase (U/L)\n* Total bilirubin (micromol/L)\n* Lactate dehydrogenase (U/L)\n* Urea (mmol/L)\n* Creatinine (micromol/L)\n* Hemoglobin (mmol/L)\n* Hematocrit (L/L)\n* Mean corpuscular volume (fL)\n* Leukocytes (10\\^9/L)\n* Thrombocytes (10\\^9/L)\n* 25-OH vitamin D (nmol/L)'}, {'measure': 'Change in psychosocial functioning', 'timeFrame': '36 months', 'description': 'Change in psychosocial functioning as estimated with the Adult Behaviour Checklist'}, {'measure': 'Change in caregiver burden', 'timeFrame': '36 months', 'description': 'Change in caregiver burden as estimated with the Zarit Burden Interview'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Prader-Willi syndrome', 'Growth hormone', 'Body composition', 'Cardiovascular disease'], 'conditions': ['Prader-Willi Syndrome']}, 'descriptionModule': {'briefSummary': 'The overall objective of this study is to measure the effect of growth hormone (GH) treatment on physical and psychosocial health in adults with Prader-Willi syndrome. Adults with PWS who have not been treated with GH during the past three years and who will start with GH treatment as part of regular patient care will be asked for informed consent to participate in this open-label prospective cohort study. We hypothesize that growth hormone treatment will improve the physical and psychosocial health.', 'detailedDescription': 'OBJECTIVES:\n\nTo measure the effect of GHt on physical and psychosocial health in adults with PWS.\n\nThe primary endpoint is change in lean body mass (LBM (kg)) as assessed by Dual Energy X-ray Absorptiometry (DEXA) scan. Secondary endpoints are total fat mass, bone density, physical health and psychosocial health. Also the occurrence of side-effects will be assessed. Only data that are collected as part of regular patient care will be used.\n\nSTUDY DESIGN:\n\nOpen-label prospective cohort study.\n\nSTUDY POPULATION:\n\nAdults with PWS who have not been treated with GH during the past three years and who will start with GHt as part of regular patient care.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Adults with PWS who have not been treated with GH during the past three years and who will start with GHt as part of regular patient care.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)\n\nExclusion Criteria:\n\n* Non cooperative behaviour\n* Pregnancy\n* Known malignancies\n* Poorly controlled diabetes (HbA1c \\> 64 mmol/mol (8%))\n* Untreated obstructive sleep apnea (apnea-hypopnea index \\> 5)\n* Body mass index above 40 kg/m2\n* Upper-airway obstruction of any cause'}, 'identificationModule': {'nctId': 'NCT04484051', 'acronym': 'GAP', 'briefTitle': 'Growth Hormone Study in Adults With Prader-Willi Syndrome', 'organization': {'class': 'OTHER', 'fullName': 'Erasmus Medical Center'}, 'officialTitle': 'Growth Hormone Study in Adults With Prader-Willi Syndroom', 'orgStudyIdInfo': {'id': 'GAP'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Routine clinical care with Genotropin treatment', 'description': 'Data collection throughout routine clinical care with subcutaneous injections Genotropin, 0.6-0.8 mg/day. Participants start with 0.2 mg/day and the dose increases with 0.2 mg/day per month to a maximum dose of 0.6-0.8 mg/day.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '3015GD', 'city': 'Rotterdam', 'state': 'South Holland', 'country': 'Netherlands', 'contacts': [{'name': 'Laura de Graaff, MD, PhD', 'role': 'CONTACT', 'email': 'l.degraaff@erasmusmc.nl', 'phone': '0031618843010'}], 'facility': 'Erasmus MC, University Medical Center Rotterdam', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}], 'centralContacts': [{'name': 'Laura de Graaff, MD, PhD', 'role': 'CONTACT', 'email': 'l.degraaff@erasmusmc.nl', 'phone': '0031618843010'}, {'name': 'Trui van Essen, MD', 'role': 'CONTACT', 'email': 'g.vanessen@erasmusmc.nl', 'phone': '0031658869674'}], 'overallOfficials': [{'name': 'Laura de Graaff, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Erasmus MC, University Medical Center Rotterdam'}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL', 'SAP'], 'timeFrame': 'The data will become available around begin 2027 (after publication of the results). The data will be available for 15 years.', 'ipdSharing': 'YES', 'description': 'The individual participant data has not been finalized yet. The investigators intent to share the full anonymized dataset, study protocol and statistical analysis plan upon request after publication of the results. Informed consent forms will not be shared.', 'accessCriteria': 'Upon reasonable request.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Erasmus Medical Center', 'class': 'OTHER'}, 'collaborators': [{'name': 'Foundation for Prader-Willi Research', 'class': 'OTHER'}, {'name': 'Prader-Willi Fonds', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'MD, PhD', 'investigatorFullName': 'dr. Laura C. G. de Graaff-Herder', 'investigatorAffiliation': 'Erasmus Medical Center'}}}}