Ignite Creation Date:
2025-12-25 @ 3:27 AM
Ignite Modification Date:
2026-02-10 @ 12:30 PM
Study NCT ID:
NCT02426905
Status:
UNKNOWN
Last Update Posted:
2017-08-23
First Post:
2014-09-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006527', 'term': 'Hepatolenticular Degeneration'}], 'ancestors': [{'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D001480', 'term': 'Basal Ganglia Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D009069', 'term': 'Movement Disorders'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008664', 'term': 'Metal Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D014266', 'term': 'Trientine'}], 'ancestors': [{'id': 'D005029', 'term': 'Ethylenediamines'}, {'id': 'D003959', 'term': 'Diamines'}, {'id': 'D011073', 'term': 'Polyamines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 90}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2016-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-08', 'completionDateStruct': {'date': '2018-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2017-08-22', 'studyFirstSubmitDate': '2014-09-18', 'studyFirstSubmitQcDate': '2015-04-24', 'lastUpdatePostDateStruct': {'date': '2017-08-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-04-27', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-05', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Clinical outcome specific to the retrospective part of the study', 'timeFrame': '48 months', 'description': "The clinical course of neurological and hepatic disease for each available time point after initiation of treatment (6, 12, 24, 36, and 48 months, and at the last available time point while taking second line trientine) will be scored (Investigator's score) based on neurological and hepatic status at the time of initiating trientine as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened."}, {'measure': 'Clinical outcome specific to the prospective part of the study', 'timeFrame': '12 months', 'description': "The clinical course of neurological and hepatic disease will be scored (Investigator's score) based on the status at 6 and 12 months after Baseline as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened A patient will be counted as a responder if they have a rating of ≤4 at the 12 month visit for both the neurological and hepatic Investigator's score. They will be counted as a non-responder if they have a rating = 5 for one or both scores at the 12 month visit or if they were discontinued from the study for any reason prior to the 12 month visit."}], 'secondaryOutcomes': [{'measure': 'Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study', 'timeFrame': 'Up to 60 months', 'description': 'All AEs related to trientine treatment, and AEs leading to discontinuation of trientine will be assessed at each available study time point.'}, {'measure': 'Quality of Life Endpoints for the Prospective Part of the Study', 'timeFrame': '12 months', 'description': 'The QoL questionnaires will be completed for each time point and data will be compared to baseline (prospective part) after 6 and 12 months'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Wilson Disease']}, 'referencesModule': {'references': [{'pmid': '40104015', 'type': 'DERIVED', 'citation': 'Mohr I, Kruse C, Aliane V, Weiss KH. Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine Dihydrochloride in Patients With Wilson Disease. JGH Open. 2025 Mar 17;9(3):e70114. doi: 10.1002/jgh3.70114. eCollection 2025 Mar.'}, {'pmid': '35482910', 'type': 'DERIVED', 'citation': 'Weiss KH, Kruse C, Manolaki N, Zuin M, Ferenci P, van Scheppingen D, Wijnberg L, de Koning CE, Dhawan A. Multicentre, retrospective study to assess long-term outcomes of chelator based treatment with trientine in Wilson disease patients withdrawn from therapy with d -penicillamine. Eur J Gastroenterol Hepatol. 2022 Sep 1;34(9):940-947. doi: 10.1097/MEG.0000000000002387. Epub 2022 Apr 29.'}]}, 'descriptionModule': {'briefSummary': 'A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.', 'detailedDescription': 'A retrospective study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them prospectively for a further 12 months.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '90 Years', 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients aged 1 year to 90 years of age.\n* Physician established diagnosis of Wilson disease based on a Ferenci score \\> 3.\n* Documented treatment with d-Penicillamine, withdrawal of treatment with d- Penicillamine, followed by treatment with trientine for at least 6 months at date of informed consent.\n* Able/willing to provide written informed consent.\n* For enrolment in the prospective part, enrolment in the retrospective part of the study is required.\n\nExclusion Criteria:\n\n* Incomplete history of medication use for trientine from initial diagnosis to latest follow up.\n* Unavailable outcome data for hepatic and neurological course of disease at assessment time points.\n* Patients with acute liver failure and fulminant hepatic disease with fatal outcome.\n* Hypersensitivity to trientine and severe anaemia.'}, 'identificationModule': {'nctId': 'NCT02426905', 'briefTitle': 'Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine', 'organization': {'class': 'INDUSTRY', 'fullName': 'Univar BV'}, 'officialTitle': 'Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine', 'orgStudyIdInfo': {'id': 'UNV-TRI-002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'NO_INTERVENTION', 'label': 'Retrospective'}, {'type': 'OTHER', 'label': 'Prospective', 'interventionNames': ['Drug: trientine dihydrochloride']}], 'interventions': [{'name': 'trientine dihydrochloride', 'type': 'DRUG', 'description': "A retrospective review of patients' medical records", 'armGroupLabels': ['Prospective']}]}, 'contactsLocationsModule': {'locations': [{'zip': '69120', 'city': 'Heidelberg', 'country': 'Germany', 'facility': 'Universitätsklinik Heidelberg', 'geoPoint': {'lat': 49.40768, 'lon': 8.69079}}, {'zip': '11527', 'city': 'Goudi', 'country': 'Greece', 'facility': '"Aghia Sophia" Children\'s Hospital', 'geoPoint': {'lat': 37.98734, 'lon': 23.76764}}, {'zip': '20142', 'city': 'Milan', 'country': 'Italy', 'facility': 'San Paolo Hospital UOC', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Kings College Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Karl-Heinz Weiss, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Universitätsklinik Heidelberg'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Univar BV', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}