Ignite Creation Date:
2025-12-25 @ 5:07 AM
Ignite Modification Date:
2025-12-26 @ 4:11 AM
Study NCT ID:
NCT03435627
Status:
COMPLETED
Last Update Posted:
2022-11-14
First Post:
2018-01-31
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009634', 'term': 'Noonan Syndrome'}], 'ancestors': [{'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D019465', 'term': 'Craniofacial Abnormalities'}, {'id': 'D009139', 'term': 'Musculoskeletal Abnormalities'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D019382', 'term': 'Human Growth Hormone'}], 'ancestors': [{'id': 'D013006', 'term': 'Growth Hormone'}, {'id': 'D010908', 'term': 'Pituitary Hormones, Anterior'}, {'id': 'D010907', 'term': 'Pituitary Hormones'}, {'id': 'D036361', 'term': 'Peptide Hormones'}, {'id': 'D006728', 'term': 'Hormones'}, {'id': 'D006730', 'term': 'Hormones, Hormone Substitutes, and Hormone Antagonists'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 71}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-02-26', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-11', 'completionDateStruct': {'date': '2022-01-18', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-11-10', 'studyFirstSubmitDate': '2018-01-31', 'studyFirstSubmitQcDate': '2018-02-09', 'lastUpdatePostDateStruct': {'date': '2022-11-14', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-02-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-01-18', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of adverse drug reactions (ADR)', 'timeFrame': 'Weeks 0-208', 'description': 'Count of events'}], 'secondaryOutcomes': [{'measure': 'Number of serious adverse drug reaction (SADR)', 'timeFrame': 'Week 0-208', 'description': 'Count of events'}, {'measure': 'Number of serious adverse event (SAE)', 'timeFrame': 'Week 0-208', 'description': 'Count of events'}, {'measure': 'Number of cardiac adverse event (AE)', 'timeFrame': 'Week 0-208', 'description': 'Count of events'}, {'measure': 'Change in insulin like growth factor I (IGF-I) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in ng/mL'}, {'measure': 'Change in insulin like growth factor I (IGF-I) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in ng/mL'}, {'measure': 'Change in insulin like growth factor I standard deviation score (IGF-I SDS) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Presented as standard deviation (SD) score'}, {'measure': 'Change in insulin like growth factor I standard deviation score (IGF-I SDS) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Presented as SD score'}, {'measure': 'Change in haemoglobin A1c (HbA1c) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in %'}, {'measure': 'Change in haemoglobin A1c (HbA1c) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in %'}, {'measure': 'Change in aspartate aminotransferase (AST) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in IU/L'}, {'measure': 'Change in aspartate aminotransferase (AST) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in IU/L'}, {'measure': 'Change in alanine aminotransferase (ALT) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in IU/L'}, {'measure': 'Change in alanine aminotransferase (ALT) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in IU/L'}, {'measure': 'Change in total cholesterol (T-CHO) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in mg/dL'}, {'measure': 'Change in total cholesterol (T-CHO) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in mg/dL'}, {'measure': 'Change in high-density lipoprotein cholesterol (HDL-C) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in mg/dL'}, {'measure': 'Change in high-density lipoprotein cholesterol (HDL-C) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in mg/dL'}, {'measure': 'Change in Low-density lipoprotein cholesterol (LDL-C) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in mg/dL'}, {'measure': 'Change in Low-density lipoprotein cholesterol (LDL-C) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in mg/dL'}, {'measure': 'Change in triglyceride (TG) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in mg/dL'}, {'measure': 'Change in triglyceride (TG) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in mg/dL'}, {'measure': 'Change in thyroid stimulation hormone(TSH) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in μU/mL'}, {'measure': 'Change in thyroid stimulation hormone(TSH) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in μU/mL'}, {'measure': 'Change in free triiodothyronine (FT-3) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in pg/mL'}, {'measure': 'Change in free triiodothyronine (FT-3) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in pg/mL'}, {'measure': 'Change in free thyroxine (FT-4) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in ng/mL'}, {'measure': 'Change in free thyroxine (FT-4) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in ng/mL'}, {'measure': 'Change in white blood cell (WBC) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in μ/L'}, {'measure': 'Change in white blood cell (WBC) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in μ/L'}, {'measure': 'Change in platelet (PLT) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in 10\\^4/μL'}, {'measure': 'Change in platelet (PLT) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in 10\\^4/μL'}, {'measure': 'Change in bone age (BA) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in year'}, {'measure': 'Change in bone age (BA) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in year'}, {'measure': 'Change in bone age/chronological age (BA/CA) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Presented as ratio'}, {'measure': 'Change in bone age/chronological age (BA/CA) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Presented as ratio'}, {'measure': 'Change in electrocardiogram (ECG) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Assessment of change in ECG is measured by categories recorded in case report forms (CRFs): normal, abnormal not clinically significant and abnormal clinically significant.'}, {'measure': 'Change in electrocardiogram (ECG) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Assessment of change in ECG is measured by categories recorded in CRFs: normal, abnormal not clinically significant and abnormal clinically significant.'}, {'measure': 'Change in height standard deviation score (HSDS) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Presented as SD score'}, {'measure': 'Change in height standard deviation score (HSDS) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Presented as SD score'}, {'measure': 'Height velocity standard deviation score (HVSDS) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Presented as SD score'}, {'measure': 'Height velocity standard deviation score (HVSDS) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Presented as SD score'}, {'measure': 'Height velocity (HV) for non-naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks', 'description': 'Measured in cm/year'}, {'measure': 'Height velocity (HV) for naïve patients', 'timeFrame': 'Weeks 0, 52, 104, 156, and 208 weeks', 'description': 'Measured in cm/year'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Genetic Disorder', 'Noonan Syndrome']}, 'referencesModule': {'references': [{'pmid': '41285480', 'type': 'DERIVED', 'citation': 'Muroya K, Kawai M, Yamagishi H, Endo T, Pietropoli A, Ferran JM, Horikawa R. Long-term effectiveness and safety of daily growth hormone therapy in Japanese children with Noonan syndrome: a post-marketing surveillance study. Endocr J. 2025 Nov 22. doi: 10.1507/endocrj.EJ25-0116. Online ahead of print.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as agreed with the study doctor.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'A total of 60 patients (consisting of non-naïve patients who were previously enrolled in study: GHLIQUID-4020 and naïve patients enrolled after approval of the Noonan indication in Japan) are planned to be included in this study.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Signed informed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).\n* The decision to initiate treatment with commercially available Norditropin® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.\n* For non-naïve patients; patients who were previously enrolled in study: GHLIQUID-4020.\n* For naïve patients; short stature due to Noonan syndrome diagnosed by the physician and a decision to initiate treatment with Norditropin® has been made by the patient/parent and the physician. At study sites, all patients will be registered consecutively from the first patient after approval date (consecutively registered system).\n* Male or female, 3 years old or over, bone age: less than 17 years old for male / less than 15 years old for female.\n\nExclusion Criteria:\n\n* Previous participation in this study. Participation is defined as having given informed consent in this study.\n* Known or suspected allergy to study products or related products.\n* In case of naïve patients, patients who have received growth hormone (GH) products for treatment of indication other than short stature due to Noonan syndrome before approval date of Noonan indication.'}, 'identificationModule': {'nctId': 'NCT03435627', 'briefTitle': 'Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novo Nordisk A/S'}, 'officialTitle': 'Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)', 'orgStudyIdInfo': {'id': 'GHLIQUID-4358'}, 'secondaryIdInfos': [{'id': 'U1111-1191-3084', 'type': 'OTHER', 'domain': 'World Health Organization (WHO)'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Norditropin® (naïve participants)', 'description': 'The treatment period of Norditropin® for naïve participants will be up to 208 weeks.', 'interventionNames': ['Drug: Somatropin']}, {'label': 'Norditropin® (non-naïve participants)', 'description': 'The treatment period of Norditropin® for non-naïve participants will be up to 442 weeks.', 'interventionNames': ['Drug: Somatropin']}], 'interventions': [{'name': 'Somatropin', 'type': 'DRUG', 'description': 'Participants will be treated with commercially available Norditropin® (somatropin) according to routine clinical practice at the discretion of the treating physician.', 'armGroupLabels': ['Norditropin® (naïve participants)', 'Norditropin® (non-naïve participants)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '078-8510', 'city': 'Asahikawa, Hokkaido', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 43.77063, 'lon': 142.36489}}, {'zip': '830-0011', 'city': 'Fukuoka', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 33.6, 'lon': 130.41667}}, {'zip': '216-8511', 'city': 'Kanagawa', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 37.58333, 'lon': 139.91667}}, {'zip': '232-8555', 'city': 'Kanagawa', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 37.58333, 'lon': 139.91667}}, {'zip': '602-8566', 'city': 'Kyoto', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 35.02107, 'lon': 135.75385}}, {'zip': '629-2261', 'city': 'Kyoto', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 35.02107, 'lon': 135.75385}}, {'zip': '371-8511', 'city': 'Maebashi-shi, Gunma', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 36.4, 'lon': 139.08333}}, {'zip': '889-1692', 'city': 'Miyazaki', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 31.91667, 'lon': 131.41667}}, {'zip': '467-8602', 'city': 'Nagoya, Aichi', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site'}, {'zip': '951 8520', 'city': 'Niigata-shi, Niigata', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site'}, {'zip': '534-0021', 'city': 'Osaka', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 34.69379, 'lon': 135.50107}}, {'zip': '594-1101', 'city': 'Osaka', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 34.69379, 'lon': 135.50107}}, {'zip': '879-5593', 'city': 'Ōita', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 33.23333, 'lon': 131.6}}, {'zip': '330-8777', 'city': 'Saitama-shi, Saitama', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 35.90807, 'lon': 139.65657}}, {'zip': '065-8611', 'city': 'Sapporo, Hokkaido', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 43.06667, 'lon': 141.35}}, {'zip': '980 8574', 'city': 'Sendai-shi, Miyagi', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site'}, {'zip': '431-3192', 'city': 'Shizuoka', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 34.98333, 'lon': 138.38333}}, {'zip': '329-0498', 'city': 'Tochigi', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 36.38333, 'lon': 139.73333}}, {'zip': '157 8535', 'city': 'Tokyo', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': '160-8582', 'city': 'Tokyo', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': '162-8666', 'city': 'Tokyo', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': '183-8561', 'city': 'Tokyo', 'country': 'Japan', 'facility': 'Novo Nordisk Investigational Site', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}], 'overallOfficials': [{'name': 'Clinical Reporting Anchor and Disclosure (1452)', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Novo Nordisk A/S'}]}, 'ipdSharingStatementModule': {'url': 'http://novonordisk-trials.com', 'ipdSharing': 'YES', 'description': 'According to the Novo Nordisk disclosure commitment on novonordisk-trials.com'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novo Nordisk A/S', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}