Ignite Creation Date:
2025-12-25 @ 2:03 AM
Ignite Modification Date:
2026-03-09 @ 4:05 AM
Study NCT ID:
NCT02228460
Status:
COMPLETED
Last Update Posted:
2019-12-17
First Post:
2014-08-27
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Czechia']}, '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': 'C000608118', 'term': 'venglustat'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Contact-US@sanofi.com', 'phone': '800-633-1610', 'title': 'Trial Transparency Team', 'phoneExt': '1#', 'organization': 'Sanofi'}, 'certainAgreement': {'otherDetails': 'The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to 212 days', 'description': 'Reported AEs are TEAEs that is AEs developed/worsened during "on treatment period" (from first administration through last administration of study drug + 30 days or end of study participation for participant, whichever occurs first). Analysis was performed on safety population that included all enrolled participants who received at least 1 dose of study drug.', 'eventGroups': [{'id': 'EG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.', 'otherNumAtRisk': 11, 'deathsNumAtRisk': 11, 'otherNumAffected': 9, 'seriousNumAtRisk': 11, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Atrial fibrillation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Blepharitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Chalazion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Eye pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Lacrimation increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Lenticular opacities', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Retinal vascular disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Vision blurred', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Oesophageal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Feeling hot', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Muscle strain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Wound', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Electrocardiogram T wave abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Electrocardiogram abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Nuclear magnetic resonance imaging brain abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Vibration test abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Muscle twitching', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Amnesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Sinus headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'White matter lesion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Depressed mood', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Angiokeratoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}], 'seriousEvents': [{'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Floppy infant', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Depressed mood', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'title': 'Baseline Score: 1 / Week 26 Score: 1', 'measurements': [{'value': '4', 'groupId': 'OG000'}]}, {'title': 'Baseline Score: 1 / Week 26 Score: 2', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': 'Baseline Score: 2 / Week 26 Score: 1', 'measurements': [{'value': '3', 'groupId': 'OG000'}]}, {'title': 'Baseline Score: 2 / Week 26 Score: 2', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.3173', 'groupIds': ['OG000'], 'pValueComment': 'Threshold for significance at 0.05 level.', 'groupDescription': 'A McNemar test was used to test the treatment effect based on paired pre-treatment and post-treatment (Week 26) frequencies of skin GL-3 score grouped into the categories (0 to \\<2; 2 to 3).', 'statisticalMethod': 'McNemar Test', 'nonInferiorityType': 'OTHER'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on full analysis set (FAS) that included all participants who received at least 1 dose of study treatment. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'PRIMARY', 'title': 'Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.22', 'groupId': 'OG000', 'lowerLimit': '-0.73', 'upperLimit': '0.29'}]}]}], 'analyses': [{'pValue': '0.625', 'groupIds': ['OG000'], 'pValueComment': 'Threshold for significance at 0.05 level.', 'groupDescription': 'Wilcoxon signed rank test was used to assess the mean change from Baseline to Week 26 in skin GL-3 score.', 'statisticalMethod': 'Wilcoxon signed rank test', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Change from Baseline in GL-3 score was obtained by subtracting Baseline value from post-baseline value at Week 26. A negative change from Baseline indicates less severe condition at Week 26.', 'unitOfMeasure': 'score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Plasma GL-3 Concentration at Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-3.62', 'spread': '1.07', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 26', 'description': 'Change from Baseline in plasma GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method.', 'unitOfMeasure': 'mcg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-30.99', 'spread': '22.83', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 26', 'description': 'Change from Baseline in plasma Lyso-GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method.', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-3.26', 'spread': '1.43', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 26', 'description': 'Change from Baseline in plasma GL-1 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method.', 'unitOfMeasure': 'mcg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'SECONDARY', 'title': 'Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'title': 'Baseline Score: 1 / Week 26 Score: 1', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': 'Baseline Score: 2 / Week 26 Score: 1', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': 'Baseline Score: 2 / Week 26 Score: 2', 'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'SECONDARY', 'title': 'Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'title': 'Baseline Score: 1.5 / Week 26 Score: 1.5', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': 'Baseline Score: 2 / Week 26 Score: 2', 'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'SECONDARY', 'title': 'Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'title': 'Baseline Score: 1 / Week 26 Score: 2', 'measurements': [{'value': '1', 'groupId': 'OG000'}]}, {'title': 'Baseline Score: 2 / Week 26 Score: 2', 'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.25', 'spread': '0.19', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 26', 'description': 'Change from Baseline in urine GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method.', 'unitOfMeasure': 'mg/mmol Cr', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26."}, {'type': 'SECONDARY', 'title': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From Baseline up to 212 days', 'description': 'Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during on-treatment period (period from the first administration of study drug through the last administration of the study drug plus 30 days or end of study participation for participant, whichever occurs first).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Analysis was performed on safety population which included all enrolled participants who received at least 1 dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics (PK): Maximum Plasma Drug Concentration (Cmax) of GZ/SAR402671', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'title': 'Day 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '24.7', 'spread': '5.89', 'groupId': 'OG000'}]}]}, {'title': 'Day 182', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '192.0', 'spread': '96.4', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)', 'description': 'Maximum plasma concentration observed for study drug was reported.', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population which included all participants for whom the primary PK data were considered sufficient and interpretable. Here, 'number analyzed' = participants with available data at specified time-point."}, {'type': 'SECONDARY', 'title': 'PK: Plasma Trough Concentration (Ctrough) of GZ/SAR402671', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'title': 'Day 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '152.0', 'spread': '68.9', 'groupId': 'OG000'}]}]}, {'title': 'Day 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '165.0', 'spread': '66.1', 'groupId': 'OG000'}]}]}, {'title': 'Day 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '182.0', 'spread': '90.3', 'groupId': 'OG000'}]}]}, {'title': 'Day 84', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '164.0', 'spread': '124.0', 'groupId': 'OG000'}]}]}, {'title': 'Day 126', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '175.0', 'spread': '94.7', 'groupId': 'OG000'}]}]}, {'title': 'Day 182', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '164.0', 'spread': '89.4', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Predose on Days 14, 28, 56, 84, 126, and 182', 'description': 'Ctrough was defined as the plasma concentration of study drug observed just before treatment administration during repeated dosing.', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point."}, {'type': 'SECONDARY', 'title': 'PK: Time to Reach Maximum Plasma Drug Concentration (Tmax) of GZ/SAR402671', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'title': 'Day 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8.00', 'groupId': 'OG000', 'lowerLimit': '1.07', 'upperLimit': '24.00'}]}]}, {'title': 'Day 182', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.00', 'groupId': 'OG000', 'lowerLimit': '0.00', 'upperLimit': '8.00'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)', 'description': 'Tmax was defined as time to reach maximum plasma concentration of study drug.', 'unitOfMeasure': 'hours', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point."}, {'type': 'SECONDARY', 'title': 'PK: Area Under Plasma Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24) of GZ/SAR402671', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'title': 'Day 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '476', 'spread': '125', 'groupId': 'OG000'}]}]}, {'title': 'Day 182', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4110', 'spread': '2090', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)', 'description': 'Area under the plasma concentration versus time curve of study drug from time 0 to 24 hours (AUC0-24) was calculated using the trapezoidal method over the dosing interval.', 'unitOfMeasure': 'ng*hour/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point."}, {'type': 'SECONDARY', 'title': 'PK: Terminal Half-life (t1/2z) of GZ/SAR402671', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'title': 'Day 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '86.8', 'spread': '39.6', 'groupId': 'OG000'}]}]}, {'title': 'Day 182', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '128.0', 'spread': '59.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)', 'description': 'Plasma t1/2z was the time measured for the plasma concentration of drug to decrease by one half. The t1/2z was estimated based on 24-hour post-dose PK.', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point."}, {'type': 'SECONDARY', 'title': 'PK: Apparent Total Body Clearance of GZ/SAR402671 at Steady State (CLss/F)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '7490', 'spread': '10900', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182', 'description': 'Apparent total body clearance at steady state was a quantitative measure of rate of clearance of drug from the blood following oral administration, and is described in terms of volume of fluid clear of drug per time unit (eg, mL/min).', 'unitOfMeasure': 'mL/hour', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure."}, {'type': 'SECONDARY', 'title': 'PK: Apparent Volume of Distribution of GZ/SAR402671 (Vss/F) at Steady State', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'timeFrame': 'Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182', 'description': 'Volume of distribution at steady state was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of drug.', 'reportingStatus': 'POSTED', 'populationDescription': 'Since the percent extrapolation of AUC for all participants was \\>30%, AUC could not be determined and hence, Vss/F could not be calculated.'}, {'type': 'SECONDARY', 'title': 'PK: Cumulated Amount of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (Ae0-24)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '3210.0', 'spread': '1640.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0-24 hours on Day 182', 'description': 'Ae0-24 was the cumulated amount of study drug excreted in urine during the time interval of 0 to 24 hours.', 'unitOfMeasure': 'mcg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure."}, {'type': 'SECONDARY', 'title': 'PK: Percentage of Dose of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (fe0-24)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '21.4', 'spread': '10.9', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0-24 hours on Day 182', 'description': 'fe0-24 was the fraction of dose excreted in urine during the time interval of 0 to 24 hours.', 'unitOfMeasure': 'percentage of dose', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure."}, {'type': 'SECONDARY', 'title': 'PK: Renal Clearance (CLR) of GZ/SAR402671 From 0 to 24 Hours', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '925', 'spread': '407', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0-24 hours on Day 182', 'description': 'CLR was calculated by dividing the cumulative amount of drug excreted in urine during the dosing interval of 0-24 hours by area under the plasma drug concentration time-curve during the dosing interval of 0-24 hours.', 'unitOfMeasure': 'mL/hour', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure."}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 milligram (mg) once daily, orally for 26 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Participants were enrolled in the study at 8 centers in 5 countries between 11 November 2014 and 06 September 2016. A total of 14 participants were screened in the study.', 'preAssignmentDetails': 'Out of 14 screened participants, 11 participants were enrolled and treated in the study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 mg once daily, orally for 26 weeks.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '26.5', 'spread': '7.6', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Analysis was performed on full analysis set (FAS) that included all participants who received at least 1 dose of study treatment.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 11}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-11', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-09', 'dispFirstSubmitDate': '2017-09-05', 'completionDateStruct': {'date': '2016-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-11-26', 'studyFirstSubmitDate': '2014-08-27', 'dispFirstSubmitQcDate': '2017-09-05', 'resultsFirstSubmitDate': '2019-11-26', 'studyFirstSubmitQcDate': '2014-08-27', 'dispFirstPostDateStruct': {'date': '2017-09-11', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2019-12-17', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-11-26', 'studyFirstPostDateStruct': {'date': '2014-08-29', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-12-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.'}, {'measure': 'Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Change from Baseline in GL-3 score was obtained by subtracting Baseline value from post-baseline value at Week 26. A negative change from Baseline indicates less severe condition at Week 26.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Plasma GL-3 Concentration at Week 26', 'timeFrame': 'Baseline, Week 26', 'description': 'Change from Baseline in plasma GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method.'}, {'measure': 'Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26', 'timeFrame': 'Baseline, Week 26', 'description': 'Change from Baseline in plasma Lyso-GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method.'}, {'measure': 'Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26', 'timeFrame': 'Baseline, Week 26', 'description': 'Change from Baseline in plasma GL-1 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method.'}, {'measure': 'Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.'}, {'measure': 'Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.'}, {'measure': 'Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26', 'timeFrame': 'Baseline, Week 26', 'description': 'Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from Baseline GL-3 score to Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.'}, {'measure': 'Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26', 'timeFrame': 'Baseline, Week 26', 'description': 'Change from Baseline in urine GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method.'}, {'measure': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs)', 'timeFrame': 'From Baseline up to 212 days', 'description': 'Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during on-treatment period (period from the first administration of study drug through the last administration of the study drug plus 30 days or end of study participation for participant, whichever occurs first).'}, {'measure': 'Pharmacokinetics (PK): Maximum Plasma Drug Concentration (Cmax) of GZ/SAR402671', 'timeFrame': 'Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)', 'description': 'Maximum plasma concentration observed for study drug was reported.'}, {'measure': 'PK: Plasma Trough Concentration (Ctrough) of GZ/SAR402671', 'timeFrame': 'Predose on Days 14, 28, 56, 84, 126, and 182', 'description': 'Ctrough was defined as the plasma concentration of study drug observed just before treatment administration during repeated dosing.'}, {'measure': 'PK: Time to Reach Maximum Plasma Drug Concentration (Tmax) of GZ/SAR402671', 'timeFrame': 'Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)', 'description': 'Tmax was defined as time to reach maximum plasma concentration of study drug.'}, {'measure': 'PK: Area Under Plasma Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24) of GZ/SAR402671', 'timeFrame': 'Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)', 'description': 'Area under the plasma concentration versus time curve of study drug from time 0 to 24 hours (AUC0-24) was calculated using the trapezoidal method over the dosing interval.'}, {'measure': 'PK: Terminal Half-life (t1/2z) of GZ/SAR402671', 'timeFrame': 'Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)', 'description': 'Plasma t1/2z was the time measured for the plasma concentration of drug to decrease by one half. The t1/2z was estimated based on 24-hour post-dose PK.'}, {'measure': 'PK: Apparent Total Body Clearance of GZ/SAR402671 at Steady State (CLss/F)', 'timeFrame': 'Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182', 'description': 'Apparent total body clearance at steady state was a quantitative measure of rate of clearance of drug from the blood following oral administration, and is described in terms of volume of fluid clear of drug per time unit (eg, mL/min).'}, {'measure': 'PK: Apparent Volume of Distribution of GZ/SAR402671 (Vss/F) at Steady State', 'timeFrame': 'Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182', 'description': 'Volume of distribution at steady state was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of drug.'}, {'measure': 'PK: Cumulated Amount of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (Ae0-24)', 'timeFrame': '0-24 hours on Day 182', 'description': 'Ae0-24 was the cumulated amount of study drug excreted in urine during the time interval of 0 to 24 hours.'}, {'measure': 'PK: Percentage of Dose of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (fe0-24)', 'timeFrame': '0-24 hours on Day 182', 'description': 'fe0-24 was the fraction of dose excreted in urine during the time interval of 0 to 24 hours.'}, {'measure': 'PK: Renal Clearance (CLR) of GZ/SAR402671 From 0 to 24 Hours', 'timeFrame': '0-24 hours on Day 182', 'description': 'CLR was calculated by dividing the cumulative amount of drug excreted in urine during the dosing interval of 0-24 hours by area under the plasma drug concentration time-curve during the dosing interval of 0-24 hours.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Venglustat'], 'conditions': ['Fabry Disease']}, 'referencesModule': {'references': [{'pmid': '36481125', 'type': 'DERIVED', 'citation': 'Deegan PB, Goker-Alpan O, Geberhiwot T, Hopkin RJ, Lukina E, Tylki-Szymanska A, Zaher A, Sensinger C, Gaemers SJM, Modur V, Thurberg BL, Sharma J, Najafian B, Mauer M, DasMahapatra P, Wilcox WR, Germain DP. Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study. Mol Genet Metab. 2023 Feb;138(2):106963. doi: 10.1016/j.ymgme.2022.11.002. Epub 2022 Nov 9.'}]}, 'descriptionModule': {'briefSummary': 'Primary Objective:\n\nTo assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.', 'detailedDescription': 'The total duration of study per participant was 7 to 8 months for participants who entered a planned extension study and approximately 13 to 14 months for participants who did not enter a planned extension study. A 2-year extension study was planned for eligible participants.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT'], 'maximumAge': '49 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion criteria:\n\n* The participant was greater than equal to (\\>=) 18 years of age and less than (\\<) 50 years of age.\n* The participant was male.\n* The participant had provided a signed informed consent.\n* The participant had a confirmed diagnosis of Fabry disease as documented by leukocyte α- Galactosidase A (αGAL) activity of \\<4 nanomole/hour/milligram (nmol/hr/mg) leukocyte (preferred assay; results from a central laboratory) or plasma αGAL \\<1.5 nanomole/hour/milliliter (nmol/hr/mL) (results from a central laboratory).\n* The participant had a plasma globotriaosylsphingosine (lyso-GL3) \\>=65 nanogram per milliliter (ng/mL).\n* The participant had never been treated with a Fabry disease-specific treatment.\n* If the participant was on renin-angiotensin-aldosterone system (RAAS) blockers and antidepressants, the dose should be stable (i.e., prescribed dose and frequency) for at least the immediate 3 months prior to screening.\n\nExclusion criteria:\n\n* The participant had an estimated glomerular filtration rate (eGFR) \\<60 mL/min/1.73 m\\^2 using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI).\n* The participant had a median urine protein/creatinine ratio (PCR) \\>=0.5 gram per gram (g/g) (median of 3 overnight urine collections. Collection of each of the 3 samples must occur between 4 and 7 days of each other, and all samples must be collected within a 15 day period). All 3 samples must be collected regardless of the results and results available prior to Day 1.\n* The participant had undergone a kidney transplant.\n* The participant had either active or a history of clinically significant organic disease (with the exception of the symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, hematologic, neurological or renal disease, or other medical condition, serious inter-current illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial.\n* The participant had abnormal liver function (serum total bilirubin \\> the upper limit of normal, or serum alanine aminotransferase (\\[ALT\\] and aspartate aminotransferase \\[AST\\] \\>2.0 times the upper limit of normal).\n* The participant had, according to World Health Organization (WHO) grading a cortical cataract (COR) \\> one-quarter of the lens circumference (Grade COR-2) or a posterior subcapsular cataract (PSC) \\>2 millimeter (mm) (Grade PSC-2). Participants with nuclear cataracts were not excluded.\n* The participant was currently receiving potentially cataractogenic medications.\n* The participant had received strong or moderate inducers or inhibitors of Cytochrome P450 3A4 (CYP3A4) per Food and Drug Administration (FDA) classification within 14 days prior to enrollment or within 5 times the elimination half-life or PD half-life of the medication, whichever is longer.\n* The participant was scheduled for in-patient hospitalization, including elective surgery, during the study.\n* The participant had a positive result on any of the following tests: hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti-HIV2 Ab). Participants with a positive hepatitis B surface antibody (HBsAb) test with a history of prior hepatitis B immunization were eligible if other criteria met (i.e., negative tests for: HBsAg, hepatitis B core antibody \\[HBcAb\\], and hepatitis C virus antibody \\[HCVAb\\]).\n* The participant had participated in a study involving an investigational drug within the past 30 days of the start of the trial.\n* The participant was unwilling to comply with the requirements of the protocol.\n* The participant was a sexually active man who was not willing to use 2 forms of birth control including a barrier method during the study until 6 weeks after the last treatment with investigational medicinal product (IMP).\n* The participant had a history or ongoing clinically significant cardiac arrhythmia, defined as either atrial fibrillation, sustained or non-sustained ventricular tachycardia.\n* The participant had any contraindication to magnetic resonance imaging (MRI).\n* The participant had one of the following central nervous system exclusion criteria:\n\n * Acute stroke, within 3 months of the screening visit.\n * History of seizures.\n\nThe above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial."}, 'identificationModule': {'nctId': 'NCT02228460', 'briefTitle': 'Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sanofi'}, 'officialTitle': 'A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease', 'orgStudyIdInfo': {'id': 'ACT13739'}, 'secondaryIdInfos': [{'id': '2013-005324-41'}, {'id': 'U1111-1152-1456', 'type': 'OTHER', 'domain': 'UTN'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'GZ/SAR402671', 'description': 'GZ/SAR402671 15 milligram (mg) once daily orally for 26 weeks.', 'interventionNames': ['Drug: GZ/SAR402671']}], 'interventions': [{'name': 'GZ/SAR402671', 'type': 'DRUG', 'description': 'Pharmaceutical form: Capsule; Route of administration: Oral', 'armGroupLabels': ['GZ/SAR402671']}]}, 'contactsLocationsModule': {'locations': [{'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Investigational Site Number 840002', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'Investigational Site Number 840003', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '22030', 'city': 'Fairfax', 'state': 'Virginia', 'country': 'United States', 'facility': 'Investigational Site Number 840001', 'geoPoint': {'lat': 38.84622, 'lon': -77.30637}}, {'zip': '92380', 'city': 'Garches', 'country': 'France', 'facility': 'Investigational Site Number 250001', 'geoPoint': {'lat': 48.84226, 'lon': 2.18232}}, {'zip': '04-730', 'city': 'Warsaw', 'country': 'Poland', 'facility': 'Investigational Site Number 616001', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'zip': '125167', 'city': 'Moscow', 'country': 'Russia', 'facility': 'Investigational Site Number 643002', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'zip': 'B15 2TH', 'city': 'Birmingham', 'country': 'United Kingdom', 'facility': 'Investigational Site Number 826003', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'CB2 OQQ', 'city': 'Cambridge', 'country': 'United Kingdom', 'facility': 'Investigational Site Number 826002', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}], 'overallOfficials': [{'name': 'Clinical Sciences & Operations', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Sanofi'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genzyme, a Sanofi Company', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}