Ignite Creation Date:
2025-12-24 @ 5:56 PM
Ignite Modification Date:
2026-02-25 @ 6:53 PM
Study NCT ID:
NCT02201368
Status:
WITHDRAWN
Last Update Posted:
2014-07-28
First Post:
2013-03-22
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C531010', 'term': 'triheptanoin'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 0}}, 'statusModule': {'whyStopped': 'IMP management difficulties', 'overallStatus': 'WITHDRAWN', 'startDateStruct': {'date': '2009-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-07', 'completionDateStruct': {'date': '2011-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-07-25', 'studyFirstSubmitDate': '2013-03-22', 'studyFirstSubmitQcDate': '2014-07-25', 'lastUpdatePostDateStruct': {'date': '2014-07-28', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2014-07-28', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of metabolic decompensation.', 'timeFrame': 'up to 16 months', 'description': 'This is a combined endpoint, including the number and/or severity of episodes of hypoglycemia, rhabdomyolysis, cardiomyopathy and liver failure after starting treatment with trihepatnoin.'}], 'secondaryOutcomes': [{'measure': 'Differences in the profiles of acylcarnitines with control.', 'timeFrame': '6 months and 6 months in each arm treatment'}, {'measure': 'Average values of transaminase and creatin kinase.', 'timeFrame': '6 months and 6 months in each arm treatment'}, {'measure': 'Differences in the fatty acid composition of plasma and red blood cells.', 'timeFrame': '6 months and 6 months in each arm treatment'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Triglycerides; Lipid Metabolism; Fatty Acids.'], 'conditions': ['Long-chain Fatty Acid Transport Deficiency']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine if administration Triheptanoin is an effective treatment for defects of the long-chain fatty acid beta-oxidation in young adults or adults. Period of treatment and follow-up will be 16 months.', 'detailedDescription': 'Triheptanoin treatment, in patients with long-chain fatty acid beta-oxidation defects, could cause not only a great improvement in their quality of life, also could prevent life-threatening signs, reducing symptoms and serious complications of their disease, like cardiomyopathy, Reye-like syndrome episodes and rhabdomyolysis. This result would occur by the effect of propionyl CoA primer on the Krebs cycle and, at the same time, would produce a gluconeogenic effect.\n\nThis treatment opens the door to be used in other diseases such as pyruvate carboxylase deficiency, glycogen storage disease and other diseases with energy problems.\n\nAll patients will be followed up until 16 months.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nAll patients with any of the following conditions:\n\n* Long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency (LCHAD).\n* Very long-chain acyl-coenzyme A dehydrogenase deficiency (VLCAD.)\n* Mitochondrial trifunctional protein (MTP).\n* Carnitine palmitoyltransferase I deficiency (CPT I).\n* Carnitine Palmitoyltransferase II (CPT II).\n* Carnitine-acylcarnitine translocase deficiency (CACT).\n\nPositive skin biopsy: patients were deemed to commence the dietary treatment with Triheptanoin after evaluating the individual response in vitro in cultured fibroblasts. This response is based on the measurement of the production of propionyl-CoA, the incubation with fatty acids odd-chain, compared with control group fibroblasts.\n\nThe informed consent must be signed by the patient or family, in the case of minors.\n\nExclusion Criteria:\n\n* No patient/family collaboration or the application of dietary treatment.\n* No in vitro test response.\n* Do not meet the inclusion criteria.'}, 'identificationModule': {'nctId': 'NCT02201368', 'briefTitle': 'Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects', 'organization': {'class': 'OTHER', 'fullName': 'Hospital Clinico Universitario de Santiago'}, 'officialTitle': 'Study to Evaluate the Effectiveness of Dietary Treatment With Triheptanoin in Patients With Long-chain Fatty Acid Beta-oxidation Defects', 'orgStudyIdInfo': {'id': '2007/084'}, 'secondaryIdInfos': [{'id': '2007-005578-29', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Triheptanoin', 'interventionNames': ['Drug: Triheptanoin (SpezialölÒ 107®)', 'Dietary Supplement: MCT (Medium-Chain Triglycerides)']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'MCT (Medium-Chain Triglycerides)', 'interventionNames': ['Drug: Triheptanoin (SpezialölÒ 107®)', 'Dietary Supplement: MCT (Medium-Chain Triglycerides)']}], 'interventions': [{'name': 'Triheptanoin (SpezialölÒ 107®)', 'type': 'DRUG', 'description': '* Randomization: Treatment with Triheptanoin for 6 months\n* Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months\n* Crossover: Treatment with MCT(Medium-Chain Triglycerides)for 6 months', 'armGroupLabels': ['MCT (Medium-Chain Triglycerides)', 'Triheptanoin']}, {'name': 'MCT (Medium-Chain Triglycerides)', 'type': 'DIETARY_SUPPLEMENT', 'description': '* Randomization: Treatment with MCT(Medium-Chain Triglycerides)for 6 months\n* Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months\n* Crossover: Treatment with Triheptanoin for 6 months', 'armGroupLabels': ['MCT (Medium-Chain Triglycerides)', 'Triheptanoin']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Santiago de Compostela', 'country': 'Spain', 'facility': 'Hospital Clínico Universitario de Santiago', 'geoPoint': {'lat': 42.88052, 'lon': -8.54569}}], 'overallOfficials': [{'name': 'Mª Luz Couce Pico', 'role': 'STUDY_CHAIR', 'affiliation': 'Hospital Clínico Universitario de Santiago'}, {'name': 'Jaume Campistol Plana', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital Sant Joan de Déu. Barcelona'}, {'name': 'Mercedes Martínez-Pardo', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital Universitario Ramon y Cajal'}, {'name': 'Mónica Ruiz Pons', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital Clínico Universitario Ntra. Sra. de Candelaria. Tenerife'}, {'name': 'Mª Teresa García Silva', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital 12 de Octubre. Madrid'}, {'name': 'Pablo Sanjurjo', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital de Cruces de Baracaldo - Bilbao'}, {'name': 'Koldo Aldamiz', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital de Cruces de Baracaldo - Bilbao'}, {'name': 'Inmaculada García Jiménez', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital Universitario Miguel Servet, Zaragoza'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Maria Luz Couce Pico', 'class': 'OTHER'}, 'collaborators': [{'name': 'Fundación Ramón Domínguez', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'MD', 'investigatorFullName': 'Maria Luz Couce Pico', 'investigatorAffiliation': 'Hospital Clinico Universitario de Santiago'}}}}