Ignite Creation Date:
2025-12-24 @ 2:58 PM
Ignite Modification Date:
2025-12-26 @ 7:32 PM
Study NCT ID:
NCT01218659
Status:
COMPLETED
Last Update Posted:
2018-11-01
First Post:
2010-10-06
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Argentina', 'Germany', 'Greece', 'Poland', 'Switzerland', 'Taiwan', 'Turkey (Türkiye)']}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'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': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C090092', 'term': 'migalastat'}, {'id': 'C525167', 'term': 'larazotide acetate'}, {'id': 'C459420', 'term': 'agalsidase beta'}, {'id': 'C000627036', 'term': 'agalsidase alfa'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'MedInfoUSA@amicusrx.com', 'phone': '+1-877-426-4287 (877-4-AMICUS)', 'title': 'Medical Affairs', 'organization': 'Amicus Therapeutics'}, 'certainAgreement': {'otherDetails': 'The investigator can publish only the results from this trial, provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review prior to submission for publication. If requested and prior to publication, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse Event (AE) data were collected from the time of signing informed consent at screening/baseline through the follow-up visit (1 mo. after the last treatment in the optional OLE/after the 18-month Randomized Period if the participant did not enroll in the OLE), for up to 30 months. Because participants in the Migalastat (0-18 mo.) and ERT (0-18 mo.) groups were combined into the All Migalastat (0-30 mo.) group, AEs for those groups are included in the All Migalastat (0-30 mo.) group AEs.', 'eventGroups': [{'id': 'EG000', 'title': 'Migalastat (0-18 Months)', 'description': 'Participants received 150 mg of migalastat orally QOD during the 18-month randomized treatment period (0-18 months). Participants received an inactive reminder capsule on alternate days during both treatment periods.', 'otherNumAtRisk': 36, 'otherNumAffected': 34, 'seriousNumAtRisk': 36, 'seriousNumAffected': 7}, {'id': 'EG001', 'title': 'ERT (0-18 Months)', 'description': "Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period (0-18 months). Participants were required to be given \\>80% of the currently labeled dose and regimen during the 18-month randomized treatment period.", 'otherNumAtRisk': 21, 'otherNumAffected': 20, 'seriousNumAtRisk': 21, 'seriousNumAffected': 7}, {'id': 'EG002', 'title': 'All Migalastat (0-30 Months)', 'description': 'Participants in this group are composed of participants from the Migalastat (0-18 month) and the ERT (0-18 month) groups. Therefore, participants in the All Migalastat group received at least 1 dose of 150 mg migalastat orally QOD during the 18-month randomized treatment period or the optional 12-month OLE (for a total time period of up to 30 months), regardless of whether or not the participant discontinued the study early, and regardless of whether they were previously randomized to migalastat or ERT during the 18-month randomized phase (0-18 months).', 'otherNumAtRisk': 51, 'otherNumAffected': 50, 'seriousNumAtRisk': 51, 'seriousNumAffected': 16}], 'otherEvents': [{'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 5}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 5}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 11}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Flatulence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 7}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 21}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 12}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Herpes Zoster', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Lower respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Blood creatine phosphokinase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 7}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Protein urine present', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'White blood cell count decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 6}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 6}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 16}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 8}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Presyncope', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 5}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 8}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Night sweats', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Neuralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Tremor', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Gastritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Procedural pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}], 'seriousEvents': [{'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Endocarditis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Perineal abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Suicidal ideation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Obesity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Device malfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Embolic stroke', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Transient ischaemic attack', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Ventricular tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Cardiac failure chronic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Atrial fibrillation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Atelectasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Bile duct stone', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Hernial eventration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Vision blurred', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Upper limb fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}, {'term': 'Phaeochromocytoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 36, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 51, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (16.1)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Migalastat (0-18 Months)', 'description': 'Participants received 150 mg migalastat orally QOD during the 18-month randomized treatment period. Participants received an inactive reminder capsule on alternate days during both treatment periods.'}, {'id': 'OG001', 'title': 'ERT (0-18 Months)', 'description': "Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given \\>80% of the currently labeled dose and regimen during the 18-month randomized treatment period. During the optional 12-month OLE period, participants received 150 mg migalastat orally QOD. Participants received an inactive reminder capsule on alternate days during the OLE."}], 'classes': [{'categories': [{'measurements': [{'value': '-4.35', 'groupId': 'OG000', 'lowerLimit': '-7.65', 'upperLimit': '-1.06'}, {'value': '-3.24', 'groupId': 'OG001', 'lowerLimit': '-7.81', 'upperLimit': '1.33'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline to Month 18', 'description': 'To assess renal function, measured glomerular filtration rate (GFR) was measured by the plasma clearance of unlabeled iohexol (mGFR-iohexol), a non-ionic contrast agent. The annualized rate of change in mGFR-iohexol from Baseline to Month 18 was analyzed using an analysis of covariance (ANCOVA) model with the following factors as covariates: treatment group, sex, age, Baseline GFR (mGFR-iohexol), and Baseline 24-hour (hr) urine protein. A threshold of \\<2.2 milliliter (mL)/minute (min)/1.73 meter squared (m\\^2)/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in estimated glomerular filtration rate (eGFR) for participants treated with agalsidase alfa for 18 months.', 'unitOfMeasure': 'mL/min/1.73 m^2/year', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation.'}, {'type': 'PRIMARY', 'title': 'Annualized Rate Of Change From Baseline To Month 18 In eGFR', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Migalastat (0-18 Months)', 'description': 'Participants received 150 mg migalastat orally QOD during the 18-month randomized treatment period. Participants received an inactive reminder capsule on alternate days during both treatment periods.'}, {'id': 'OG001', 'title': 'ERT (0-18 Months)', 'description': "Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given \\>80% of the currently labeled dose and regimen during the 18-month randomized treatment period. During the optional 12-month OLE period, participants received 150 mg migalastat orally QOD. Participants received an inactive reminder capsule on alternate days during the OLE."}], 'classes': [{'categories': [{'measurements': [{'value': '-0.40', 'groupId': 'OG000', 'lowerLimit': '-2.27', 'upperLimit': '1.48'}, {'value': '-1.03', 'groupId': 'OG001', 'lowerLimit': '-3.64', 'upperLimit': '1.58'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline to Month 18', 'description': 'The eGFR assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation was calculated using the following:\n\neGFR-CKD-EPI = 141 x min (Serum Creatinine/κ,1)\\^(α) x max(Serum Creatinine/κ,1)\\^(-1.209) x 0.993\\^(Age) x 1.1018 (if female) x 1.159 (if African American or black) where: κ is 0.7 for females and 0.9 for males; α is -0.329 for females and -0.411 for males; min indicates the minimum of Serum Creatinine/κ or 1; max indicates the maximum of Serum Creatinine/κ or 1.\n\nThe annualized rate of change in eGFR-CKD-EPI from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. A threshold of \\<2.2 mL/min/1.73m\\^2/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in eGFR for participants treated with agalsidase alfa for 18 months.', 'unitOfMeasure': 'mL/min/1.73 m^2/year', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation.'}, {'type': 'SECONDARY', 'title': 'Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Migalastat (0-18 Months)', 'description': 'Participants received 150 mg migalastat orally QOD during the 18-month randomized treatment period. Participants received an inactive reminder capsule on alternate days during both treatment periods.'}, {'id': 'OG001', 'title': 'ERT (0-18 Months)', 'description': "Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given \\>80% of the currently labeled dose and regimen during the 18-month randomized treatment period. During the optional 12-month OLE period, participants received 150 mg migalastat orally QOD. Participants received an inactive reminder capsule on alternate days during the OLE."}], 'classes': [{'categories': [{'measurements': [{'value': '-1.51', 'groupId': 'OG000', 'lowerLimit': '-3.43', 'upperLimit': '0.40'}, {'value': '-1.53', 'groupId': 'OG001', 'lowerLimit': '-4.20', 'upperLimit': '1.13'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline to Month 18', 'description': "The GFR estimated by the Modification Of Diet In Renal Disease equation (eGFR-MDRD) was calculated using the following equation: eGFR-MDRD = 175 x (Serum Creatinine)\\^(-1.154) x (Age)\\^(-0.203) x 1.212 (if participant's race is black or African American) x 0.742 (if participant is female).\n\nThe eGFR-MDRD from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein.", 'unitOfMeasure': 'mL/min/1.73 m^2/year', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Migalastat (0-18 Months)', 'description': 'Participants received 150 milligrams (mg) of migalastat hydrochloride (migalastat) (equivalent to 123 mg of migalastat) orally once every other day (QOD) during the 18-month randomized treatment period (0-18 months). Participants received an inactive reminder capsule on alternate days during both treatment periods.'}, {'id': 'FG001', 'title': 'ERT (0-18 Months)', 'description': "Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period (0-18 months). Participants were required to be given \\>80% of the currently labeled dose and regimen during the 18-month randomized treatment period."}, {'id': 'FG002', 'title': 'All Migalastat (0-30 Months)', 'description': 'Participants in this group are composed of participants from the Migalastat (0-18 months) and the ERT (0-18 months) groups. Therefore, participants in the All Migalastat group received at least 1 dose of 150 mg migalastat orally QOD during the 18-month randomization period or the optional 12-month OLE (for a total time period of up to 30 months), regardless of whether or not the participant discontinued the study early, and regardless of whether they were previously randomized to migalastat or ERT during the 18-month Randomized Period (0-18 months).'}], 'periods': [{'title': '18-Month Randomized Period', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '36'}, {'groupId': 'FG001', 'numSubjects': '24'}, {'comment': 'Zero participants are noted here because participants started in this group during the 12-month OLE.', 'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Safety Population', 'comment': 'Safety: received at least 1 dose of migalastat or ERT.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '36'}, {'groupId': 'FG001', 'numSubjects': '21'}, {'comment': 'Zero participants are noted here because participants started in this group during the 12-month OLE.', 'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}, {'groupId': 'FG001', 'numSubjects': '18'}, {'comment': 'Zero participants are noted here because participants started in this group during the 12-month OLE.', 'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '6'}, {'groupId': 'FG002', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'Did not receive study drug', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}, {'title': '12-Month OLE', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'Zero participants are noted here because they were combined in the All Migalastat (0-30 mo.) group.', 'groupId': 'FG000', 'numSubjects': '0'}, {'comment': 'Zero participants are noted here because they were combined in the All Migalastat (0-30 mo.) group.', 'groupId': 'FG001', 'numSubjects': '0'}, {'comment': 'Fifty-two participants completed the 18-mo. randomized treatment period.', 'groupId': 'FG002', 'numSubjects': '52'}]}, {'type': 'Safety Population', 'comment': 'Safety: ITT pop. who received ≥1 dose of study drug during the 18-mo randomized treatment period.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '51'}]}, {'type': 'Modified Intent-to-Treat (mITT)', 'comment': 'mITT: Eligibility based on Good Laboratory Practice (GLP) human embryonic kidney cell (HEK) assay.', 'achievements': [{'comment': 'Zero participants are noted here because they were combined in the All Migalastat (0-30 mo.) group.', 'groupId': 'FG000', 'numSubjects': '0'}, {'comment': 'Zero participants are noted here because they were combined in the All Migalastat (0-30 mo.) group.', 'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '46'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Zero participants are noted here because they were combined in the All Migalastat (0-30 mo.) group.', 'groupId': 'FG000', 'numSubjects': '0'}, {'comment': 'Zero participants are noted here because they were combined in the All Migalastat (0-30 mo.) group.', 'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '42'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '10'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '2'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Pregnancy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Lack of Efficacy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}, {'type': 'Declined to participate in the OLE', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '4'}]}]}], 'preAssignmentDetails': 'The All Migalastat group was comprised of participants who received migalastat during the randomized treatment period (0-18 mo.) and who received enzyme replacement therapy (ERT) during the randomized treatment period (0-18 mo.) and switched to migalastat during the 12-mo. open-label extension (OLE). 8 participants enrolled but did not randomize.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'BG000'}, {'value': '21', 'groupId': 'BG001'}, {'value': '57', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Migalastat (0-18 Months)', 'description': 'Participants received 150 mg migalastat orally QOD during the 18-month randomized treatment period. Participants received an inactive reminder capsule on alternate days during both treatment periods.'}, {'id': 'BG001', 'title': 'ERT (0-18 Months)', 'description': "Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given \\>80% of the currently labeled dose and regimen during the 18-month randomized treatment period. Participants received an inactive reminder capsule on alternate days during the OLE."}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '50.5', 'spread': '13.76', 'groupId': 'BG000'}, {'value': '46.3', 'spread': '14.91', 'groupId': 'BG001'}, {'value': '48.9', 'spread': '14.21', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '20', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '32', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Safety Population: All randomized participants who received at least 1 dose of migalastat or ERT.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 68}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-09-08', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-10', 'dispFirstSubmitDate': '2015-06-25', 'completionDateStruct': {'date': '2015-05-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-10-01', 'studyFirstSubmitDate': '2010-10-06', 'dispFirstSubmitQcDate': '2015-06-25', 'resultsFirstSubmitDate': '2018-08-10', 'studyFirstSubmitQcDate': '2010-10-08', 'dispFirstPostDateStruct': {'date': '2015-07-20', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2018-11-01', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-10-01', 'studyFirstPostDateStruct': {'date': '2010-10-11', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-11-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2014-05-27', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate', 'timeFrame': 'Baseline to Month 18', 'description': 'To assess renal function, measured glomerular filtration rate (GFR) was measured by the plasma clearance of unlabeled iohexol (mGFR-iohexol), a non-ionic contrast agent. The annualized rate of change in mGFR-iohexol from Baseline to Month 18 was analyzed using an analysis of covariance (ANCOVA) model with the following factors as covariates: treatment group, sex, age, Baseline GFR (mGFR-iohexol), and Baseline 24-hour (hr) urine protein. A threshold of \\<2.2 milliliter (mL)/minute (min)/1.73 meter squared (m\\^2)/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in estimated glomerular filtration rate (eGFR) for participants treated with agalsidase alfa for 18 months.'}, {'measure': 'Annualized Rate Of Change From Baseline To Month 18 In eGFR', 'timeFrame': 'Baseline to Month 18', 'description': 'The eGFR assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation was calculated using the following:\n\neGFR-CKD-EPI = 141 x min (Serum Creatinine/κ,1)\\^(α) x max(Serum Creatinine/κ,1)\\^(-1.209) x 0.993\\^(Age) x 1.1018 (if female) x 1.159 (if African American or black) where: κ is 0.7 for females and 0.9 for males; α is -0.329 for females and -0.411 for males; min indicates the minimum of Serum Creatinine/κ or 1; max indicates the maximum of Serum Creatinine/κ or 1.\n\nThe annualized rate of change in eGFR-CKD-EPI from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. A threshold of \\<2.2 mL/min/1.73m\\^2/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in eGFR for participants treated with agalsidase alfa for 18 months.'}], 'secondaryOutcomes': [{'measure': 'Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation', 'timeFrame': 'Baseline to Month 18', 'description': "The GFR estimated by the Modification Of Diet In Renal Disease equation (eGFR-MDRD) was calculated using the following equation: eGFR-MDRD = 175 x (Serum Creatinine)\\^(-1.154) x (Age)\\^(-0.203) x 1.212 (if participant's race is black or African American) x 0.742 (if participant is female).\n\nThe eGFR-MDRD from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein."}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Amicus Therapeutics', 'Galafold', 'AT1001', 'Migalastat'], 'conditions': ['Fabry Disease']}, 'referencesModule': {'references': [{'pmid': '33012654', 'type': 'DERIVED', 'citation': 'Feldt-Rasmussen U, Hughes D, Sunder-Plassmann G, Shankar S, Nedd K, Olivotto I, Ortiz D, Ohashi T, Hamazaki T, Skuban N, Yu J, Barth JA, Nicholls K. Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study. Mol Genet Metab. 2020 Sep-Oct;131(1-2):219-228. doi: 10.1016/j.ymgme.2020.07.007. Epub 2020 Aug 15.'}, {'pmid': '32994552', 'type': 'DERIVED', 'citation': 'Bichet DG, Aerts JM, Auray-Blais C, Maruyama H, Mehta AB, Skuban N, Krusinska E, Schiffmann R. Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease. Genet Med. 2021 Jan;23(1):192-201. doi: 10.1038/s41436-020-00968-z. Epub 2020 Sep 30.'}, {'pmid': '31934472', 'type': 'DERIVED', 'citation': 'Haninger-Vacariu N, El-Hadi S, Pauler U, Foretnik M, Kain R, Prohaszka Z, Schmidt A, Skuban N, Barth JA, Sunder-Plassmann G. Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report. Case Rep Obstet Gynecol. 2019 Dec 21;2019:1030259. doi: 10.1155/2019/1030259. eCollection 2019.'}, {'pmid': '31889231', 'type': 'DERIVED', 'citation': 'Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.'}, {'pmid': '27834756', 'type': 'DERIVED', 'citation': 'Hughes DA, Nicholls K, Shankar SP, Sunder-Plassmann G, Koeller D, Nedd K, Vockley G, Hamazaki T, Lachmann R, Ohashi T, Olivotto I, Sakai N, Deegan P, Dimmock D, Eyskens F, Germain DP, Goker-Alpan O, Hachulla E, Jovanovic A, Lourenco CM, Narita I, Thomas M, Wilcox WR, Bichet DG, Schiffmann R, Ludington E, Viereck C, Kirk J, Yu J, Johnson F, Boudes P, Benjamin ER, Lockhart DJ, Barlow C, Skuban N, Castelli JP, Barth J, Feldt-Rasmussen U. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. J Med Genet. 2017 Apr;54(4):288-296. doi: 10.1136/jmedgenet-2016-104178. Epub 2016 Nov 10.'}, {'pmid': '27657681', 'type': 'DERIVED', 'citation': 'Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22.'}]}, 'descriptionModule': {'briefSummary': 'Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the clinical trial human embryonic kidney cell (HEK) assay.', 'detailedDescription': "This was a Phase 3, randomized, open-label, active-controlled study to evaluate the efficacy and safety of 150 milligrams (mg) of migalastat hydrochloride (migalastat) (equivalent to 123 mg of migalastat) once every other day (QOD) and ERT in male and female participants with Fabry disease who were receiving ERT and who have an α Gal-A mutation that is amenable to migalastat, based on the clinical trial HEK assay. This was a 2-part study. Part 1, the 18-month randomized phase, evaluated participants who received either migalastat 150 mg QOD or ERT per prescribing physicians' instructions for efficacy and safety. Part 2, the optional 12-month open-label extension (OLE) phase in which all participants received migalastat, also explored efficacy and safety. For Part 2, all participants who received ERT in Part 1 were given migalastat. Data presented in this posting include efficacy data from the 18-month randomized period and safety data from the entire study (18-month randomized period and 12-month optional OLE \\[total of 30 months\\])."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '74 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female between the ages of 16 and 74 diagnosed with Fabry disease\n* Confirmed α Gal-A mutation that is amenable to migalastat, based on the clinical trial HEK assay\n* Participant has been on ERT for at least 12 months before screening/baseline\n* Dose level and regimen of ERT have been stable for 3 months before screening/baseline and is at least 80% of the currently labeled dose and regimen for this time period\n* Glomerular filtration rate (GFR) ≥ 30 milliliter (mL)/minute (min) /1.73 m\\^2\n* Participants taking angiotensin converting enzyme inhibitors or angiotensin receptor blockers must be on a stable dose for at least 4 weeks before screening/baseline\n* Women who can become pregnant and all men agree to be sexually abstinent or use medically accepted methods of birth control throughout the duration of the study and for up to 30 days after last dose of study medication\n* Participant is willing and able to provide written informed consent and assent if applicable\n\nExclusion Criteria:\n\n* Participant has undergone, or is scheduled to undergo, kidney transplantation or any other solid organ transplantation\n* Participant is on regular dialysis that is specifically for the treatment of chronic kidney disease\n* Participant has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before screening/baseline\n* Participant has clinically significant unstable cardiac disease in the opinion of the investigator (for example, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association (NYHA) class III or IV congestive heart failure)\n* Pregnant or breast-feeding\n* History of allergy or sensitivity to study medication (including excipients) or other iminosugars (for example, miglustat, miglitol)\n* Participant has absolute contraindication to iohexol and/or inability to undergo iohexol GFR testing\n* Participant requires treatment with Glyset® (miglitol), or Zavesca® (miglustat)\n* Participant received any investigational/experimental drug, biologic or device within 30 days of screening/baseline\n* Any intercurrent illness or condition that may preclude the participant from fulfilling the study requirements or suggests to the investigator that the participant may have an unacceptable risk by participating in this study'}, 'identificationModule': {'nctId': 'NCT01218659', 'briefTitle': 'Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Amicus Therapeutics'}, 'officialTitle': 'A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT', 'orgStudyIdInfo': {'id': 'AT1001-012'}, 'secondaryIdInfos': [{'id': 'ATTRACT', 'type': 'OTHER', 'domain': 'Amicus Therapeutics'}, {'id': '2010-022636-37', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Migalastat', 'description': 'Participants received 150 mg migalastat orally QOD during the 18-month randomized treatment period and the optional 12-month OLE period. Participants received an inactive reminder capsule on alternate days during both treatment periods.', 'interventionNames': ['Drug: migalastat hydrochloride']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'ERT', 'description': "Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given \\>80% of the currently labeled dose and regimen during the 18-month randomized treatment period. During the optional 12-month OLE period, participants received 150 mg migalastat orally QOD. Participants received an inactive reminder capsule on alternate days during the OLE.", 'interventionNames': ['Biological: agalsidase']}], 'interventions': [{'name': 'migalastat hydrochloride', 'type': 'DRUG', 'otherNames': ['AT1001', 'Migalastat', 'Galafold'], 'description': '150-mg capsule administered orally QOD', 'armGroupLabels': ['Migalastat']}, {'name': 'agalsidase', 'type': 'BIOLOGICAL', 'otherNames': ['agalsidase beta; Fabrazyme', 'agalsidase alfa; Replagal'], 'description': "Agalsidase via intravenous infusion as prescribed by the participant's treating physician and in accordance with the approved prescribing information", 'armGroupLabels': ['ERT']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90048', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '30033', 'city': 'Decatur', 'state': 'Georgia', 'country': 'United States', 'geoPoint': {'lat': 33.77483, 'lon': -84.29631}}, {'zip': '49525', 'city': 'Grand Rapids', 'state': 'Michigan', 'country': 'United States', 'geoPoint': {'lat': 42.96336, 'lon': -85.66809}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '22030', 'city': 'Fairfax', 'state': 'Virginia', 'country': 'United States', 'geoPoint': {'lat': 38.84622, 'lon': -77.30637}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '03050', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': '6000', 'city': 'Perth', 'state': 'Western Australia', 'country': 'Australia', 'geoPoint': {'lat': -31.95224, 'lon': 115.8614}}, {'zip': '1090', 'city': 'Vienna', 'country': 'Austria', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'zip': '2650', 'city': 'Edegem', 'country': 'Belgium', 'geoPoint': {'lat': 51.15662, 'lon': 4.44504}}, {'zip': '14048-900', 'city': 'São Paulo', 'country': 'Brazil', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'zip': '2100', 'city': 'Copenhagen', 'country': 'Denmark', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'zip': '92380', 'city': 'Garches', 'country': 'France', 'geoPoint': {'lat': 48.84226, 'lon': 2.18232}}, {'zip': '59037', 'city': 'Lille', 'country': 'France', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'zip': '50129', 'city': 'Florence', 'country': 'Italy', 'geoPoint': {'lat': 43.77925, 'lon': 11.24626}}, {'zip': '951-8520', 'city': 'Niigata', 'country': 'Japan', 'geoPoint': {'lat': 37.92259, 'lon': 139.04125}}, {'zip': '545-8586', 'city': 'Osaka', 'country': 'Japan', 'geoPoint': {'lat': 34.69379, 'lon': 135.50107}}, {'zip': '565-0871', 'city': 'Osaka', 'country': 'Japan', 'geoPoint': {'lat': 34.69379, 'lon': 135.50107}}, {'zip': '105-8471', 'city': 'Tokyo', 'country': 'Japan', 'geoPoint': {'lat': 35.6895, 'lon': 139.69171}}, {'zip': 'CB2 0QQ', 'city': 'Cambridge', 'country': 'United Kingdom', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'zip': 'NW3 2QG', 'city': 'London', 'country': 'United Kingdom', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'WC1N 3BG', 'city': 'London', 'country': 'United Kingdom', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'M6 8HD', 'city': 'Salford', 'country': 'United Kingdom', 'geoPoint': {'lat': 53.48771, 'lon': -2.29042}}], 'overallOfficials': [{'name': 'Medical Monitor, Clinical Research', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Amicus Therapeutics'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Amicus Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}