Ignite Creation Date:
2025-12-25 @ 2:08 AM
Ignite Modification Date:
2026-02-28 @ 3:48 AM
Study NCT ID:
NCT01165060
Status:
COMPLETED
Last Update Posted:
2011-08-10
First Post:
2010-07-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000326', 'term': 'Adrenoleukodystrophy'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D018901', 'term': 'Peroxisomal Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000309', 'term': 'Adrenal Insufficiency'}, {'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001629', 'term': 'Bezafibrate'}], 'ancestors': [{'id': 'D001549', 'term': 'Benzamides'}, {'id': 'D000577', 'term': 'Amides'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D058607', 'term': 'Fibric Acids'}, {'id': 'D058610', 'term': 'Isobutyrates'}, {'id': 'D002087', 'term': 'Butyrates'}, {'id': 'D000144', 'term': 'Acids, Acyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D001565', 'term': 'Benzoates'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002723', 'term': 'Chlorobenzoates'}, {'id': 'D010647', 'term': 'Phenyl Ethers'}, {'id': 'D004987', 'term': 'Ethers'}, {'id': 'D001555', 'term': 'Benzene Derivatives'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D010636', 'term': 'Phenols'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 10}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-07'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2010-07', 'completionDateStruct': {'date': '2011-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2011-08-09', 'studyFirstSubmitDate': '2010-07-13', 'studyFirstSubmitQcDate': '2010-07-16', 'lastUpdatePostDateStruct': {'date': '2011-08-10', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2010-07-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Very long chain fatty acids (VLCFA; C22:0, C24:0 and C26:0) in plasma, lymphocytes and erythrocytes.', 'timeFrame': '24 weeks'}], 'secondaryOutcomes': [{'measure': 'Side effects', 'timeFrame': 'At 4, 8, 12, 16, 20 and 24 weeks.'}, {'measure': 'Cholesterol (total-, HDL, and LDL-cholesterol) in plasma.', 'timeFrame': '24 weeks'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['X-ALD', 'AMN', 'adrenomyeloneuropathy phenotype'], 'conditions': ['X-linked Adrenoleukodystrophy', 'Adrenomyeloneuropathy']}, 'referencesModule': {'references': [{'pmid': '22911730', 'type': 'DERIVED', 'citation': 'Engelen M, Tran L, Ofman R, Brennecke J, Moser AB, Dijkstra IM, Wanders RJ, Poll-The BT, Kemp S. Bezafibrate for X-linked adrenoleukodystrophy. PLoS One. 2012;7(7):e41013. doi: 10.1371/journal.pone.0041013. Epub 2012 Jul 20.'}]}, 'descriptionModule': {'briefSummary': 'X-linked adrenoleukodystrophy (X-ALD) is an inherited metabolic disorder characterised by accumulation of very long chain fatty acids (VLCFA) in plasma and tissue. Presumably this accumulation is responsible for tissue damage. The disease can cause severe demyelinisation of the central nervous system usually causing death in childhood or progressive ambulatory problems in adults caused by a progressive myelopathy. For the latter category of patients no curative treatment is currently available. Recent investigations in human fibroblasts and mice identified bezafibrate as an agent that might reduce VLCFA in patients with X-ALD.\n\nObjective of the study:\n\nThe trial is designed as an open-label pilot study. The main goal is to investigate if bezafibrate can reduce VLCFA in vivo in patient with X-ALD. If there is indeed a biochemical effect, a large follow-up study will be initiated with clinical outcome parameters.\n\nStudy design:\n\n10 men with X-ALD will use bezafibrate during a period of 6 months (in combination with a low fat diet). On 6 different time points the participants will undergo a venipuncture for detecting possible side effects and to determine the biochemical outcome parameters.\n\nStudy population:\n\nAdult men with X-linked adrenoleukodystrophy.\n\nIntervention (if applicable):\n\nBezafibrate.\n\nPrimary study parameters/outcome of the study:\n\nThe primary outcome parameters are cholesterol levels (total-, LDL, and HDL) and levels of triglycerides in plasma, VLCFA levels in plasma, leukocytes and erythrocytes and also C26:0-lyso-PC in bloodspots.\n\nSecondary study parameters/outcome of the study (if applicable):\n\nSecondary outcome parameters are side-effects (subjective and abnormalities in the safety lab).'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* an age of 18 years or older\n* capable of giving informed consent and capable of visiting the hospital for follow-up visits\n* no contra-indications for the use of bezafibrate, e.g. kidney- and/or liver disease.\n* confirmed X-ALD, AMN phenotype (confirmed by VLCFA analysis or analysis of the ABCD1 gene)\n\nExclusion Criteria:\n\n* use of medication that lowers cholesterol and/or triglycerides (e.g. statins)\n* liver disease or and increase in serum CK of more than 3 times the baseline level\n* treatment with Lorenzo's oil in the 8 weeks preceding the trial"}, 'identificationModule': {'nctId': 'NCT01165060', 'acronym': 'BEZA', 'briefTitle': 'The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)', 'organization': {'class': 'OTHER', 'fullName': 'Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)'}, 'officialTitle': 'Effect of Bezafibrate on Very Long Chain Fatty Acid Metabolism in Men With X-linked Adrenoleukodystrophy (X-ALD)', 'orgStudyIdInfo': {'id': 'MEC 09/278'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Bezafibrate', 'description': 'All patients in the trial will use bezafibrate 400 mg once daily until week 12, and subsequently use 800 mg onde daily until week 24.', 'interventionNames': ['Drug: Bezafibrate']}], 'interventions': [{'name': 'Bezafibrate', 'type': 'DRUG', 'otherNames': ['Bezalip retard.'], 'description': 'Week 0 to 12: 400 mg once daily 1 tablet. Week 13 to 24: 400 mg once daily 2 tablets.', 'armGroupLabels': ['Bezafibrate']}]}, 'contactsLocationsModule': {'locations': [{'zip': '1100 DD', 'city': 'Amsterdam', 'state': 'North Holland', 'country': 'Netherlands', 'facility': 'Academisch Medisch Centrum', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}], 'overallOfficials': [{'name': 'Bwee Tien Poll - The, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)', 'class': 'OTHER'}, 'collaborators': [{'name': 'The Stop ALD Foundation', 'class': 'UNKNOWN'}], 'responsibleParty': {'oldNameTitle': 'Department of (pediatric) neurology', 'oldOrganization': 'Academisch Medisch Centrum, Amsterdam, The Netherlands'}}}}