Ignite Creation Date:
2025-12-25 @ 2:09 AM
Ignite Modification Date:
2025-12-26 @ 4:48 PM
Study NCT ID:
NCT01495260
Status:
COMPLETED
Last Update Posted:
2019-03-11
First Post:
2011-11-28
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants
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': 'D000111', 'term': 'Acetylcysteine'}, {'id': 'D008063', 'term': 'Thioctic Acid'}, {'id': 'D014810', 'term': 'Vitamin E'}], 'ancestors': [{'id': 'D003545', 'term': 'Cysteine'}, {'id': 'D000603', 'term': 'Amino Acids, Sulfur'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D013876', 'term': 'Thiophenes'}, {'id': 'D003067', 'term': 'Coenzymes'}, {'id': 'D045762', 'term': 'Enzymes and Coenzymes'}, {'id': 'D005227', 'term': 'Fatty Acids'}, {'id': 'D008055', 'term': 'Lipids'}, {'id': 'D001578', 'term': 'Benzopyrans'}, {'id': 'D011714', 'term': 'Pyrans'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 13}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-09', 'completionDateStruct': {'date': '2013-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-03-07', 'studyFirstSubmitDate': '2011-11-28', 'studyFirstSubmitQcDate': '2011-12-16', 'lastUpdatePostDateStruct': {'date': '2019-03-11', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2011-12-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'oxidative lesion biomarkers', 'timeFrame': '12 months', 'description': 'oxidative lesion biomarkers: protein, DNA and peroxidation biomarkers'}], 'secondaryOutcomes': [{'measure': 'clinical parameters', 'timeFrame': '2, 6 and 12 months', 'description': 'spasticity, disability,electroneurograms and evocated potentials. Cranial and spinal NMR will be done at the beginning and the end of the trial.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['XALD', 'AMN'], 'conditions': ['Adrenomyeloneuropathy']}, 'referencesModule': {'references': [{'pmid': '21786300', 'type': 'BACKGROUND', 'citation': 'Lopez-Erauskin J, Fourcade S, Galino J, Ruiz M, Schluter A, Naudi A, Jove M, Portero-Otin M, Pamplona R, Ferrer I, Pujol A. Antioxidants halt axonal degeneration in a mouse model of X-adrenoleukodystrophy. Ann Neurol. 2011 Jul;70(1):84-92. doi: 10.1002/ana.22363.'}, {'pmid': '21453200', 'type': 'BACKGROUND', 'citation': 'Galino J, Ruiz M, Fourcade S, Schluter A, Lopez-Erauskin J, Guilera C, Jove M, Naudi A, Garcia-Arumi E, Andreu AL, Starkov AA, Pamplona R, Ferrer I, Portero-Otin M, Pujol A. Oxidative damage compromises energy metabolism in the axonal degeneration mouse model of X-adrenoleukodystrophy. Antioxid Redox Signal. 2011 Oct 15;15(8):2095-107. doi: 10.1089/ars.2010.3877. Epub 2011 Jun 8.'}, {'pmid': '35778568', 'type': 'DERIVED', 'citation': 'Parameswaran J, Goicoechea L, Planas-Serra L, Pastor A, Ruiz M, Calingasan NY, Guilera C, Aso E, Boada J, Pamplona R, Portero-Otin M, de la Torre R, Ferrer I, Casasnovas C, Pujol A, Fourcade S. Activating cannabinoid receptor 2 preserves axonal health through GSK-3beta/NRF2 axis in adrenoleukodystrophy. Acta Neuropathol. 2022 Aug;144(2):241-258. doi: 10.1007/s00401-022-02451-2. Epub 2022 Jul 1.'}, {'pmid': '31077039', 'type': 'DERIVED', 'citation': 'Casasnovas C, Ruiz M, Schluter A, Naudi A, Fourcade S, Veciana M, Castaner S, Alberti A, Bargallo N, Johnson M, Raymond GV, Fatemi A, Moser AB, Villarroya F, Portero-Otin M, Artuch R, Pamplona R, Pujol A. Biomarker Identification, Safety, and Efficacy of High-Dose Antioxidants for Adrenomyeloneuropathy: a Phase II Pilot Study. Neurotherapeutics. 2019 Oct;16(4):1167-1182. doi: 10.1007/s13311-019-00735-2.'}], 'seeAlsoLinks': [{'url': 'http://www.neurometabolic-lab.org/', 'label': 'The Neurometabolic Diseases Lab, led by ICREA Research Professor Aurora Pujol, is integrated in the Neurosciences Area of IDIBELL.'}, {'url': 'http://www.idibell.cat/', 'label': 'Related Info'}, {'url': 'http://www.bellvitgehospital.cat/', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': 'X-linked adrenoleukodystrophy is a rare, demyelinating and neurodegenerative disorder, due to a loss of function of a fatty acid transporter, the peroxisomal ABCD1protein. Its more frequent phenotype, the adrenomyeloneuropathy in adults, is characterized by axonal degeneration in spinal cord, spastic paraparesis and a disabling peripheral neuropathy. Actually, there is no efficient treatment for the disease. Our work in the last twelve years dissecting the physiopathological basis of the disorder has uncovered an involvement of the oxidative stress early in the neurodegenerative cascade. In a preclinical trial we have identified an antioxidant cocktail that efficiently reverse the clinical symptoms and the axonal degeneration in the mouse model for the disease. We propose the translation of the results to an open trial to test the tolerance and effectiveness of these drugs in the correction of the previously identified oxidative lesion biomarkers, as a first step to a randomized versus placebo, multicentric and international trial. You will be clinically explored and assessed in the Hospital Universitari of Bellvitge (HUB) using clinical scales for spasticity, disability, electroneurogram and cranial and spinal Nuclear Magnetic resonance (NMR). The information will be collected in a data base that will be of great value to improve the present attention and the future follow-up to facilitate your inclusion in therapeutic randomized, double blind, against placebo clinical trials.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '64 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Symptomatic AMN patients,\n* 18-64 years old,\n* male and female,\n* clinically and biochemically diagnosed;\n* females must be obligated heterozygotes or must have gene mutation identified.\n\nExclusion Criteria:\n\n* Pregnant and lactation in females,\n* Cerebral inflammatory disease with cognitive disorder, and/or\n* need the help of two walking sticks,\n* epilepsy,\n* hypersensibility to cysteine related compounds,\n* transaminases 2 fold up normal values.'}, 'identificationModule': {'nctId': 'NCT01495260', 'briefTitle': 'A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants', 'organization': {'class': 'INDIV', 'fullName': 'Pujol, Aurora, M.D.'}, 'officialTitle': 'A Clinical Trial for Adrenomyeloneuropathy (AMN): Validation of Biomarkers of Oxidative Stress, and Efficacy, Tolerance and Safety of a Mixture of the Antioxidants N-acetylcysteine, Lipoic Acid and Vitamin E', 'orgStudyIdInfo': {'id': 'XAMNANTIOXAP2010'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'N-acetylcysteine, lipoic acid and vitamin E', 'description': 'Two Dose titration design', 'interventionNames': ['Drug: N-acetylcysteine', 'Drug: lipoic acid', 'Drug: vitamin E']}], 'interventions': [{'name': 'N-acetylcysteine', 'type': 'DRUG', 'description': 'N-acetylcysteine, 800-2400 mg daily for 2 months', 'armGroupLabels': ['N-acetylcysteine, lipoic acid and vitamin E']}, {'name': 'lipoic acid', 'type': 'DRUG', 'description': 'lipoic acid, 300-600 mg daily for 2 months', 'armGroupLabels': ['N-acetylcysteine, lipoic acid and vitamin E']}, {'name': 'vitamin E', 'type': 'DRUG', 'description': 'vitamin E, 150-300 mg daily for 2 months', 'armGroupLabels': ['N-acetylcysteine, lipoic acid and vitamin E']}]}, 'contactsLocationsModule': {'locations': [{'zip': '08907', 'city': "L'Hospitalet de Llobregat", 'state': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Universitari de Bellvitge', 'geoPoint': {'lat': 41.35967, 'lon': 2.10028}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pujol, Aurora, M.D.', 'class': 'INDIV'}, 'collaborators': [{'name': 'Ministerio de Sanidad, Servicios Sociales e Igualdad', 'class': 'OTHER_GOV'}, {'name': 'Fundacion Hesperia', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}