Ignite Creation Date:
2025-12-24 @ 5:24 PM
Ignite Modification Date:
2025-12-27 @ 10:25 PM
Study NCT ID:
NCT00358150
Status:
COMPLETED
Last Update Posted:
2017-02-15
First Post:
2006-07-27
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D005776', 'term': 'Gaucher 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': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'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': 'C522917', 'term': 'eliglustat'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Contact-us@sanofi.com', 'title': 'Trial Transparency Team', 'organization': 'Sanofi'}, 'certainAgreement': {'otherDetails': 'If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Due to the very small number of participants at Year 9, the point estimates of mean and standard deviation are not reliable.'}}, 'adverseEventsModule': {'timeFrame': 'Up to Year 9.', 'description': 'Safety set: participants who signed informed consent, received at least 1 dose. Data reported as per dose groups (50/100 mg) as well as for all participants. In the event, 1 participant experienced both serious and non-serious forms of same AE, individual was included in numerator (number of participants affected) of each AE table.', 'eventGroups': [{'id': 'EG000', 'title': 'Eliglustat 50 mg BID', 'description': 'Participants who received eliglustat capsule as single 50 mg dose on Day 1 followed by eliglustat 50 mg BID from Day 2 to Year 9.', 'otherNumAtRisk': 5, 'otherNumAffected': 5, 'seriousNumAtRisk': 5, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Eliglustat 100 mg BID', 'description': 'Participants who received eliglustat capsule as single 50 mg dose on Day 1 followed by eliglustat 50 mg capsule BID from Day 2 to Day 19 and then eliglustat 100 mg capsule BID from Day 20 to Year 9.', 'otherNumAtRisk': 18, 'otherNumAffected': 18, 'seriousNumAtRisk': 18, 'seriousNumAffected': 3}, {'id': 'EG002', 'title': 'Eliglustat', 'description': 'Participants who received eliglustat capsule as a single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID or eliglustat 100 mg BID from Day 20 to Year 9. Participants who discontinued prior to Day 20 dose had their dose group set to missing and are only included in the all participants group. The Eliglustat group contains all participants who were treated with Eliglustat, including 2 participants dosed with 50 mg QD and 1 participant dosed with 150 mg BID for majority of study. Participants who had dose adjustment will be presented in dose group they received for majority of study.', 'otherNumAtRisk': 26, 'otherNumAffected': 23, 'seriousNumAtRisk': 26, 'seriousNumAffected': 5}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Bone marrow disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 8}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Lymphadenopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Splenomegaly', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Diastolic dysfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Left atrial dilatation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Ear haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Autoimmune thyroiditis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dental caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Food poisoning', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Haemorrhoids', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Hiatus hernia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Odynophagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Oesophagitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rectal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Regurgitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Chills', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Peripheral swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Vessel puncture site swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Biliary dyskinesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Bacteriuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Infected dermal cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Onychomycosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tooth infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 5}, 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'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Atrophic vulvovaginitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Benign prostatic hyperplasia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cervical dysplasia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cervical polyp', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Erectile dysfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Genital pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Metrorrhagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Paranasal sinus discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dermal cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dermatitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dry skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Ecchymosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Prurigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Purpura', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rosacea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Telangiectasia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tooth extraction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}], 'seriousEvents': [{'term': 'Ventricular tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Inguinal hernia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cholecystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Exposure during pregnancy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Abortion spontaneous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Ovarian cyst ruptured', 'stats': [{'groupId': 'EG000', 'numAtRisk': 5, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 18, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 26, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants Demonstrating A Meaningful Clinical Response', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'categories': [{'measurements': [{'value': '77', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline, Year 1', 'description': 'A meaningful clinical response was defined as an improvement in at least 2 of the 3 main efficacy parameters: a) an increase in hemoglobin of greater than or equal to (\\>=) 0.5 gram/deciliter from baseline, b) an increase in platelets of \\>=15 percent (%) from baseline, c) reduction in total spleen volume of \\>= 15% from baseline. As hemoglobin, platelets, total spleen volume were abnormal at baseline, within each participant, only those parameters were used in the evaluation of meaningful clinical response which were abnormal at baseline.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat.'}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=22)', 'categories': [{'measurements': [{'value': '-38.5', 'spread': '11.41', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n=20)', 'categories': [{'measurements': [{'value': '-52.4', 'spread': '10.73', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 3 (n=19)', 'categories': [{'measurements': [{'value': '-59.1', 'spread': '11.68', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 4 (n=18)', 'categories': [{'measurements': [{'value': '-62.5', 'spread': '11.63', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 5 (n=19)', 'categories': [{'measurements': [{'value': '-63.9', 'spread': '14.64', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 6 (n=19)', 'categories': [{'measurements': [{'value': '-66.2', 'spread': '15.72', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 7 (n=19)', 'categories': [{'measurements': [{'value': '-67.8', 'spread': '15.65', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 8 (n=15)', 'categories': [{'measurements': [{'value': '-67.9', 'spread': '17.11', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '-52.6', 'spread': '23.97', 'groupId': 'OG000'}]}]}, {'title': 'Change at End of Study (n=19)', 'categories': [{'measurements': [{'value': '-68.6', 'spread': '18.60', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Percent change in spleen volume = (\\[spleen volume at specified time points minus spleen volume at baseline\\] divided by \\[spleen volume at baseline\\]) multiplied by 100, where all volumes are in multiples of normal.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=22)', 'categories': [{'measurements': [{'value': '-16.9', 'spread': '10.48', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n=20)', 'categories': [{'measurements': [{'value': '-23.9', 'spread': '12.81', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 3 (n=19)', 'categories': [{'measurements': [{'value': '-26.8', 'spread': '12.28', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 4 (n=18)', 'categories': [{'measurements': [{'value': '-28.0', 'spread': '13.80', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 5 (n=19)', 'categories': [{'measurements': [{'value': '-31.2', 'spread': '13.90', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 6 (n=19)', 'categories': [{'measurements': [{'value': '-28.4', 'spread': '23.58', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 7 (n=19)', 'categories': [{'measurements': [{'value': '-36.2', 'spread': '13.49', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 8 (n=15)', 'categories': [{'measurements': [{'value': '-31.1', 'spread': '13.51', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '-22.0', 'spread': '18.49', 'groupId': 'OG000'}]}]}, {'title': 'Change at End of Study (n=19)', 'categories': [{'measurements': [{'value': '-36.4', 'spread': '14.91', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Percent change in liver volume = (\\[liver volume at specified time points minus liver volume at baseline\\] divided by \\[liver volume at baseline\\]) multiplied by 100, where all volumes are in multiples of normal.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=22)', 'categories': [{'measurements': [{'value': '1.70', 'spread': '1.274', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n=20)', 'categories': [{'measurements': [{'value': '2.13', 'spread': '1.507', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 3 (n=18)', 'categories': [{'measurements': [{'value': '2.47', 'spread': '1.406', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 4 (n=19)', 'categories': [{'measurements': [{'value': '2.27', 'spread': '1.451', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 5 (n=19)', 'categories': [{'measurements': [{'value': '2.09', 'spread': '1.746', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 6 (n=18)', 'categories': [{'measurements': [{'value': '2.01', 'spread': '1.326', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 7 (n=19)', 'categories': [{'measurements': [{'value': '2.07', 'spread': '1.485', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 8 (n=16)', 'categories': [{'measurements': [{'value': '2.08', 'spread': '1.748', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '1.00', 'spread': '1.480', 'groupId': 'OG000'}]}]}, {'title': 'Change at End of Study (n=18)', 'categories': [{'measurements': [{'value': '2.01', 'spread': '1.785', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Absolute change = hemoglobin level at specified time points minus hemoglobin level at baseline.', 'unitOfMeasure': 'g/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=22)', 'categories': [{'measurements': [{'value': '41.3', 'spread': '36.95', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n=20)', 'categories': [{'measurements': [{'value': '81.5', 'spread': '56.01', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 3 (n=18)', 'categories': [{'measurements': [{'value': '87.9', 'spread': '65.37', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 4 (n=19)', 'categories': [{'measurements': [{'value': '95.1', 'spread': '89.41', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 5 (n=19)', 'categories': [{'measurements': [{'value': '90.9', 'spread': '85.42', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 6 (n=17)', 'categories': [{'measurements': [{'value': '114.3', 'spread': '102.48', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 7 (n=19)', 'categories': [{'measurements': [{'value': '99.4', 'spread': '97.13', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 8 (n=16)', 'categories': [{'measurements': [{'value': '109.8', 'spread': '114.73', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 9 (n=3)', 'categories': [{'measurements': [{'value': '45.4', 'spread': '56.28', 'groupId': 'OG000'}]}]}, {'title': 'Change at End of Study (n=18)', 'categories': [{'measurements': [{'value': '117.5', 'spread': '116.91', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Percent change in platelet count = (\\[platelet count at specified time points minus platelet count at baseline\\] divided by \\[platelet count at baseline\\]) multiplied by 100.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=22)', 'categories': [{'measurements': [{'value': '-35.1', 'spread': '18.87', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n=20)', 'categories': [{'measurements': [{'value': '-53.5', 'spread': '21.05', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 3 (n=19)', 'categories': [{'measurements': [{'value': '-56.7', 'spread': '19.33', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 4 (n=18)', 'categories': [{'measurements': [{'value': '-60.7', 'spread': '18.44', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 5 (n=19)', 'categories': [{'measurements': [{'value': '-55.7', 'spread': '22.51', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 6 (n=17)', 'categories': [{'measurements': [{'value': '-61.5', 'spread': '24.73', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 7 (n=19)', 'categories': [{'measurements': [{'value': '-63.6', 'spread': '24.64', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 8 (n=16)', 'categories': [{'measurements': [{'value': '-59.2', 'spread': '21.77', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '-55.1', 'spread': '32.58', 'groupId': 'OG000'}]}]}, {'title': 'Change at End of Study (n=18)', 'categories': [{'measurements': [{'value': '-64.7', 'spread': '23.38', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time point."}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=22)', 'categories': [{'measurements': [{'value': '-37.0', 'spread': '11.64', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n= 10)', 'categories': [{'measurements': [{'value': '-52.5', 'spread': '10.61', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points. As per the change in planned analysis, TRAP was not assessed after Year 2."}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=21)', 'categories': [{'measurements': [{'value': '-49.0', 'spread': '24.89', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n=15)', 'categories': [{'measurements': [{'value': '-72.1', 'spread': '19.11', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 3 (n=18)', 'categories': [{'measurements': [{'value': '-68.6', 'spread': '17.27', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 4 (n=18)', 'categories': [{'measurements': [{'value': '-80.2', 'spread': '12.71', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 5 (n=18)', 'categories': [{'measurements': [{'value': '-89.4', 'spread': '8.48', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 6 (n=18)', 'categories': [{'measurements': [{'value': '-82.7', 'spread': '11.98', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 7 (n=18)', 'categories': [{'measurements': [{'value': '-78.5', 'spread': '17.83', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 8 (n=16)', 'categories': [{'measurements': [{'value': '-79.8', 'spread': '16.19', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '-88.6', 'spread': '6.30', 'groupId': 'OG000'}]}]}, {'title': 'Change at End of Study (n=18)', 'categories': [{'measurements': [{'value': '-85.8', 'spread': '12.92', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=20)', 'categories': [{'measurements': [{'value': '-49.9', 'spread': '17.17', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n=18)', 'categories': [{'measurements': [{'value': '-73.5', 'spread': '14.93', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 3 (n=17)', 'categories': [{'measurements': [{'value': '-76.4', 'spread': '12.78', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 4 (n=17)', 'categories': [{'measurements': [{'value': '-79.2', 'spread': '16.18', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 5 (n=17)', 'categories': [{'measurements': [{'value': '-76.7', 'spread': '19.16', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 6 (n=17)', 'categories': [{'measurements': [{'value': '-74.1', 'spread': '26.97', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 7 (n=17)', 'categories': [{'measurements': [{'value': '-75.5', 'spread': '27.88', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 8 (n=14)', 'categories': [{'measurements': [{'value': '-72.5', 'spread': '29.20', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '-61.2', 'spread': '36.45', 'groupId': 'OG000'}]}]}, {'title': 'Change at End of Study (n=17)', 'categories': [{'measurements': [{'value': '-69.8', 'spread': '36.59', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Physical Functioning: Change at Year 1 (n=22)', 'categories': [{'measurements': [{'value': '8.41', 'spread': '14.670', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Change at Year 2 (n=20)', 'categories': [{'measurements': [{'value': '9.50', 'spread': '17.837', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Change at Year 3 (n=17)', 'categories': [{'measurements': [{'value': '12.65', 'spread': '18.296', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Change at Year 4 (n=19)', 'categories': [{'measurements': [{'value': '11.32', 'spread': '16.231', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Change at Year 5 (n=19)', 'categories': [{'measurements': [{'value': '10.76', 'spread': '19.169', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Change at Year 6 (n=19)', 'categories': [{'measurements': [{'value': '12.63', 'spread': '17.589', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Change at Year 7 (n=19)', 'categories': [{'measurements': [{'value': '11.58', 'spread': '17.955', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Change at Year 8 (n=16)', 'categories': [{'measurements': [{'value': '11.56', 'spread': '17.485', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '1.25', 'spread': '2.500', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning:Change at End of Study (n=19)', 'categories': [{'measurements': [{'value': '9.28', 'spread': '17.085', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 1 (n=22)', 'categories': [{'measurements': [{'value': '4.55', 'spread': '22.091', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 2 (n=20)', 'categories': [{'measurements': [{'value': '5.31', 'spread': '27.824', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 3 (n=17)', 'categories': [{'measurements': [{'value': '9.56', 'spread': '24.717', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 4 (n=19)', 'categories': [{'measurements': [{'value': '6.58', 'spread': '26.391', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 5 (n=19)', 'categories': [{'measurements': [{'value': '7.57', 'spread': '28.227', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 6 (n=19)', 'categories': [{'measurements': [{'value': '8.88', 'spread': '25.964', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 7 (n=19)', 'categories': [{'measurements': [{'value': '7.89', 'spread': '25.331', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 8 (n=16)', 'categories': [{'measurements': [{'value': '8.59', 'spread': '29.393', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '-1.56', 'spread': '18.663', 'groupId': 'OG000'}]}]}, {'title': 'Role - Physical: Change at End of Study (n= 19)', 'categories': [{'measurements': [{'value': '10.53', 'spread': '24.211', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 1 (n= 22)', 'categories': [{'measurements': [{'value': '-0.77', 'spread': '21.307', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 2 (n= 20)', 'categories': [{'measurements': [{'value': '4.40', 'spread': '25.050', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 3 (n= 17)', 'categories': [{'measurements': [{'value': '4.24', 'spread': '21.297', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 4 (n= 19)', 'categories': [{'measurements': [{'value': '-2.53', 'spread': '25.202', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 5 (n= 19)', 'categories': [{'measurements': [{'value': '0.37', 'spread': '23.104', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 6 (n= 19)', 'categories': [{'measurements': [{'value': '-2.32', 'spread': '26.268', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 7 (n= 19)', 'categories': [{'measurements': [{'value': '1.74', 'spread': '27.777', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 8 (n= 16)', 'categories': [{'measurements': [{'value': '1.44', 'spread': '25.532', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '1.00', 'spread': '19.630', 'groupId': 'OG000'}]}]}, {'title': 'Bodily Pain: Change at End of Study (n= 19)', 'categories': [{'measurements': [{'value': '6.11', 'spread': '29.603', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 1 (n= 22)', 'categories': [{'measurements': [{'value': '10.36', 'spread': '19.822', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 2 (n= 20)', 'categories': [{'measurements': [{'value': '11.30', 'spread': '24.262', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 3 (n= 17)', 'categories': [{'measurements': [{'value': '16.94', 'spread': '23.485', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 4 (n= 19)', 'categories': [{'measurements': [{'value': '15.53', 'spread': '23.136', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 5 (n= 19)', 'categories': [{'measurements': [{'value': '13.58', 'spread': '23.710', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 6 (n= 19)', 'categories': [{'measurements': [{'value': '12.83', 'spread': '23.443', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 7 (n= 19)', 'categories': [{'measurements': [{'value': '15.89', 'spread': '23.278', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 8 (n= 16)', 'categories': [{'measurements': [{'value': '18.19', 'spread': '20.140', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '11.25', 'spread': '11.087', 'groupId': 'OG000'}]}]}, {'title': 'General Health: Change at End of Study (n= 19)', 'categories': [{'measurements': [{'value': '15.00', 'spread': '21.422', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 1 (n= 22)', 'categories': [{'measurements': [{'value': '5.68', 'spread': '17.242', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 2 (n= 20)', 'categories': [{'measurements': [{'value': '7.71', 'spread': '26.127', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 3 (n= 17)', 'categories': [{'measurements': [{'value': '14.71', 'spread': '21.188', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 4 (n= 19)', 'categories': [{'measurements': [{'value': '10.20', 'spread': '20.005', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 5 (n= 19)', 'categories': [{'measurements': [{'value': '13.82', 'spread': '23.623', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 6 (n= 19)', 'categories': [{'measurements': [{'value': '12.50', 'spread': '23.936', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 7 (n= 19)', 'categories': [{'measurements': [{'value': '7.57', 'spread': '22.782', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 8 (n= 16)', 'categories': [{'measurements': [{'value': '15.23', 'spread': '23.493', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '3.13', 'spread': '10.825', 'groupId': 'OG000'}]}]}, {'title': 'Vitality: Change at End of Study (n= 19)', 'categories': [{'measurements': [{'value': '15.13', 'spread': '21.278', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 1 (n= 22)', 'categories': [{'measurements': [{'value': '-0.57', 'spread': '23.298', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 2 (n= 20)', 'categories': [{'measurements': [{'value': '10.00', 'spread': '26.470', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 3 (n= 17)', 'categories': [{'measurements': [{'value': '9.56', 'spread': '19.024', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 4 (n= 19)', 'categories': [{'measurements': [{'value': '3.29', 'spread': '25.291', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 5 (n= 19)', 'categories': [{'measurements': [{'value': '9.21', 'spread': '23.140', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 6 (n= 19)', 'categories': [{'measurements': [{'value': '9.21', 'spread': '26.628', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 7 (n= 19)', 'categories': [{'measurements': [{'value': '8.55', 'spread': '20.435', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 8 (n= 16)', 'categories': [{'measurements': [{'value': '11.72', 'spread': '24.778', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '3.13', 'spread': '6.250', 'groupId': 'OG000'}]}]}, {'title': 'Social Functioning: Change at End of Study (n= 19)', 'categories': [{'measurements': [{'value': '9.21', 'spread': '25.291', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 1 (n= 22)', 'categories': [{'measurements': [{'value': '-3.03', 'spread': '17.733', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 2 (n= 20)', 'categories': [{'measurements': [{'value': '4.58', 'spread': '20.675', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 3 (n= 17)', 'categories': [{'measurements': [{'value': '2.45', 'spread': '16.342', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 4 (n= 19)', 'categories': [{'measurements': [{'value': '3.95', 'spread': '18.916', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 5 (n= 19)', 'categories': [{'measurements': [{'value': '2.19', 'spread': '22.020', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 6 (n= 19)', 'categories': [{'measurements': [{'value': '3.07', 'spread': '23.110', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 7 (n= 19)', 'categories': [{'measurements': [{'value': '0.44', 'spread': '23.485', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 8 (n= 16)', 'categories': [{'measurements': [{'value': '5.21', 'spread': '19.927', 'groupId': 'OG000'}]}]}, {'title': 'Role - Emotional: Change at Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '-10.42', 'spread': '12.501', 'groupId': 'OG000'}]}]}, {'title': 'Role- Emotional: Change at End of study (n= 19)', 'categories': [{'measurements': [{'value': '1.32', 'spread': '22.952', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 1 (n= 22)', 'categories': [{'measurements': [{'value': '0.23', 'spread': '17.827', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 2 (n= 20)', 'categories': [{'measurements': [{'value': '1.75', 'spread': '19.485', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 3 (n=17)', 'categories': [{'measurements': [{'value': '7.65', 'spread': '16.117', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 4 (n= 19)', 'categories': [{'measurements': [{'value': '4.21', 'spread': '19.809', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 5 (n= 19)', 'categories': [{'measurements': [{'value': '6.84', 'spread': '19.945', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 6 (n= 19)', 'categories': [{'measurements': [{'value': '5.53', 'spread': '22.785', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 7 (n= 19)', 'categories': [{'measurements': [{'value': '6.05', 'spread': '21.186', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 8 (n= 16)', 'categories': [{'measurements': [{'value': '7.66', 'spread': '19.989', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '3.75', 'spread': '4.787', 'groupId': 'OG000'}]}]}, {'title': 'Mental Health: Change at End of Study (n=19)', 'categories': [{'measurements': [{'value': '8.68', 'spread': '20.196', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary:Change at Year 1(n= 22)', 'categories': [{'measurements': [{'value': '3.58', 'spread': '6.263', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary:Change at Year 2(n= 20)', 'categories': [{'measurements': [{'value': '3.81', 'spread': '7.968', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary:Change at Year 3(n= 17)', 'categories': [{'measurements': [{'value': '5.17', 'spread': '7.044', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary:Change at Year 4(n= 19)', 'categories': [{'measurements': [{'value': '3.69', 'spread': '8.195', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary:Change at Year 5(n= 19)', 'categories': [{'measurements': [{'value': '3.75', 'spread': '8.430', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary:Change at Year 6(n= 19)', 'categories': [{'measurements': [{'value': '3.88', 'spread': '6.038', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary:Change at Year 7(n= 19)', 'categories': [{'measurements': [{'value': '4.57', 'spread': '7.892', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary:Change at Year 8(n= 16)', 'categories': [{'measurements': [{'value': '4.36', 'spread': '6.946', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary: Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '1.98', 'spread': '5.029', 'groupId': 'OG000'}]}]}, {'title': 'Physical Component Summary: Change at EOS (n= 19)', 'categories': [{'measurements': [{'value': '4.70', 'spread': '7.120', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 1 (n= 22)', 'categories': [{'measurements': [{'value': '-1.03', 'spread': '8.703', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 2 (n= 20)', 'categories': [{'measurements': [{'value': '2.04', 'spread': '10.476', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 3 (n= 17)', 'categories': [{'measurements': [{'value': '3.45', 'spread': '7.881', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 4 (n= 19)', 'categories': [{'measurements': [{'value': '2.11', 'spread': '10.013', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 5 (n= 19)', 'categories': [{'measurements': [{'value': '3.50', 'spread': '10.963', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 6 (n= 19)', 'categories': [{'measurements': [{'value': '3.03', 'spread': '12.535', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 7 (n= 19)', 'categories': [{'measurements': [{'value': '1.94', 'spread': '10.982', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 8 (n= 16)', 'categories': [{'measurements': [{'value': '4.59', 'spread': '10.106', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '-0.52', 'spread': '1.287', 'groupId': 'OG000'}]}]}, {'title': 'Mental Component Summary: Change at EOS (n=19)', 'categories': [{'measurements': [{'value': '3.73', 'spread': '11.243', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': "The SF-36 questionnaire, version 2, investigates the participant's health-related quality of life (HRQL). It is a 36-item questionnaire measuring 8 domains (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life. Two summary scale scores were computed from the 8 domain scores: the Physical Component Summary and the Mental Component Summary. Score range for both summary scale ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Change at Year 1 (n=17)', 'categories': [{'measurements': [{'value': '-0.56', 'spread': '1.169', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 2 (n=16)', 'categories': [{'measurements': [{'value': '-0.63', 'spread': '1.388', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 3 (n=14)', 'categories': [{'measurements': [{'value': '-1.37', 'spread': '1.983', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 4 (n=16)', 'categories': [{'measurements': [{'value': '-1.41', 'spread': '1.574', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 5 (n=16)', 'categories': [{'measurements': [{'value': '-1.26', 'spread': '1.418', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 6 (n=15)', 'categories': [{'measurements': [{'value': '-1.21', 'spread': '1.681', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 7 (n=16)', 'categories': [{'measurements': [{'value': '-0.97', 'spread': '1.429', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 8 (n=13)', 'categories': [{'measurements': [{'value': '-1.22', 'spread': '1.489', 'groupId': 'OG000'}]}]}, {'title': 'Change at Year 9 (n=4)', 'categories': [{'measurements': [{'value': '-1.20', 'spread': '1.192', 'groupId': 'OG000'}]}]}, {'title': 'Change at End of Study (n=16)', 'categories': [{'measurements': [{'value': '-1.16', 'spread': '1.665', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'The FSS is an instrument consisting of 9 self-administered questions that measures the impact of severity of fatigue symptoms on everyday functioning, based on the recall over the past week. Score range for each question ranges from 1 (minimum) to 7 (maximum), where higher score indicates greater severity. FSS total score was calculated by averaging the results of all questions. Total FSS score ranges from 9 (minimum) to 63 (maximum), where higher scores indicates greater severity.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'None: Baseline (n=26)', 'categories': [{'measurements': [{'value': '23', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 1 (n=22)', 'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 2 (n=20)', 'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 3 (n=19)', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 4 (n=19)', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 5 (n=19)', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 6 (n=19)', 'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 7 (n=19)', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 8 (n=16)', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'None: Year 9 (n=4)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'None: End of Study (n=19)', 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Baseline (n=26)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 1 (n=22)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 2 (n=20)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 3 (n=19)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 4 (n=19)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 5 (n=19)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 6 (n=19)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 7 (n=19)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 8 (n=16)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: Year 9 (n=4)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Very Mild: End of Study (n=19)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Baseline (n= 26)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 1 (n= 22)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 2 (n= 20)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 3 (n=19)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 4 (n=19)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 5 (n=19)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 6 (n=19)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 7 (n=19)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 8 (n=16)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild: Year 9 (n=4)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Mild: End of Study (n= 19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Baseline (n=26)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 1 (n=22)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 2 (n=20)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 3 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 4 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 5 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 6 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 7 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 8 (n=16)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: Year 9 (n=4)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Moderate: End of Study (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain and moderate bone pain. In this outcome, number of participants with different levels of bone pain at specified time points were reported.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'MS: Unrestricted Mobility: Baseline (n=26)', 'categories': [{'measurements': [{'value': '24', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 1 (n=22)', 'categories': [{'measurements': [{'value': '22', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 2 (n=20)', 'categories': [{'measurements': [{'value': '20', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 3 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 4 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 5 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 6 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 7 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 8 (n=16)', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: Year 9 (n=4)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'MS: Unrestricted Mobility: End of Study (n=19)', 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Baseline (n=26)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 1 (n=22)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 2 (n=20)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 3 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 4 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 5 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 6 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 7 (n=19)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 8 (n=16)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: Year 9 (n=4)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'MS: Walks with Difficulty: End of Study (n=19)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Mobillity, i.e. ability to walk was assessed as a part of Gaucher disease assessment in participants.In this outcome, number of participants with their different mobility status (unrestricted mobility, walks with difficulty) at specified time points were reported.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Baseline (n=26)', 'categories': [{'measurements': [{'value': '26', 'groupId': 'OG000'}]}]}, {'title': 'Year 1 (n=22)', 'categories': [{'measurements': [{'value': '22', 'groupId': 'OG000'}]}]}, {'title': 'Year 2 (n=20)', 'categories': [{'measurements': [{'value': '20', 'groupId': 'OG000'}]}]}, {'title': 'Year 3 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Year 4 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Year 5 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Year 6 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Year 7 (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Year 8 (n=16)', 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': 'Year 9 (n=4)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'End of Study (n=19)', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with 0= no bone crises levels at specified time points were reported.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 8. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Femoral Head: Improved: Year 1: (n=16)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Femoral Head: Improved: Year 2: (n=14)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Femoral Head: Improved: Year 3: (n=13)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Femoral Head: Improved: Year 4: (n=13)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Femoral Head: Improved: Year 5: (n=13)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Femoral Head: Improved: Year 6: (n=12)', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Femoral Head: Improved: Year 7: (n=13)', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Femoral Head: Improved: Year 8: (n=13)', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Femoral Head: Improved: End of Study: (n=8)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: Year 1 (n=8)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: Year 2 (n=7)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: Year 3 (n=6)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: Year 4 (n=6)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: Year 5 (n=7)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: Year 6 (n=6)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: Year 7 (n=7)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: Year 8 (n=7)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Greater Trochanter: Improved: End of Study (n=4)', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: Year 1 (n=21)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: Year 2 (n=19)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: Year 3 (n=18)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: Year 4 (n=18)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: Year 5 (n=18)', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: Year 6 (n=17)', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: Year 7 (n=18)', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: Year 8 (n=17)', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Intertrochanteric Regions: Improved: EOS (n=12)', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: Year 1 (n= 20)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: Year 2 (n= 18)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: Year 3 (n= 17)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: Year 4 (n= 17)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: Year 5 (n= 17)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: Year 6 (n= 16)', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: Year 7 (n= 17)', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: Year 8 (n= 16)', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Shaft: Improved: End of Study (n=11)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: Year 1 (n=21)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: Year 2 (n=19)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: Year 3 (n=18)', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: Year 4 (n=18)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: Year 5 (n=18)', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: Year 6 (n=17)', 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: Year 7 (n=18)', 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: Year 8 (n=17)', 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'Distal Metaphysis: Improved: End of Study (n=12)', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: Year 1 (n=11)', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: Year 2 (n=9)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: Year 3 (n=8)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: Year 4 (n=8)', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: Year 5 (n=8)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: Year 6 (n=7)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: Year 7 (n=8)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: Year 8 (n=8)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Condyles: Improved: End of Study (n=5)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (up to Year 9)', 'description': 'Bone marrow infiltration assessments were designed to evaluate improvements in dark marrow using MRI. Each MRI assessment was performed for both femurs and consisted of reviewing 6 different zones (the femoral head, greater trochanter, intertrochanteric region, shaft, distal metaphysis, and condyles). MRI images recorded dark marrow for each zone as either present or not present at baseline. In this outcome, number of participants (for whom dark marrow was present at baseline) with improvement from baseline in dark marrow at each specified time point were reported.', 'calculatePct': False, 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants who were presented with dark marrow at baseline and had data available at specified time points."}, {'type': 'SECONDARY', 'title': 'Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Lumbar Spine T-Score: Baseline (n= 25)', 'categories': [{'measurements': [{'value': '-1.85', 'spread': '1.094', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 1 (n= 20)', 'categories': [{'measurements': [{'value': '-1.43', 'spread': '1.015', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 2 (n= 17)', 'categories': [{'measurements': [{'value': '-0.93', 'spread': '1.268', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 3 (n= 15)', 'categories': [{'measurements': [{'value': '-1.09', 'spread': '1.143', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 4 (n= 15)', 'categories': [{'measurements': [{'value': '-0.88', 'spread': '1.258', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 5 (n= 15)', 'categories': [{'measurements': [{'value': '-0.79', 'spread': '1.116', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 6 (n= 15)', 'categories': [{'measurements': [{'value': '-0.69', 'spread': '1.313', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 7 (n= 15)', 'categories': [{'measurements': [{'value': '-0.59', 'spread': '1.265', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 8 (n= 14)', 'categories': [{'measurements': [{'value': '-0.59', 'spread': '1.294', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '0.00', 'spread': '0.753', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine T-Score: End of Study (n= 6)', 'categories': [{'measurements': [{'value': '-0.35', 'spread': '1.613', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Baseline (n= 23)', 'categories': [{'measurements': [{'value': '-0.47', 'spread': '0.894', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 1 (n= 19)', 'categories': [{'measurements': [{'value': '-0.24', 'spread': '0.954', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 2 (n= 15)', 'categories': [{'measurements': [{'value': '-0.09', 'spread': '0.991', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 3 (n= 13)', 'categories': [{'measurements': [{'value': '0.16', 'spread': '0.987', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 4 (n= 13)', 'categories': [{'measurements': [{'value': '0.13', 'spread': '1.044', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 5 (n= 13)', 'categories': [{'measurements': [{'value': '0.14', 'spread': '1.016', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 6 (n= 13)', 'categories': [{'measurements': [{'value': '0.19', 'spread': '0.946', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 7 (n= 13)', 'categories': [{'measurements': [{'value': '0.12', 'spread': '1.021', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 8 (n= 12)', 'categories': [{'measurements': [{'value': '0.33', 'spread': '0.907', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '0.33', 'spread': '0.618', 'groupId': 'OG000'}]}]}, {'title': 'Femur T-Scores: End of Study (n= 6)', 'categories': [{'measurements': [{'value': '0.18', 'spread': '1.278', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)', 'description': "Images of the lumbar spine and femur were obtained by dual energy X-ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-scores compares participant's bone density with that of healthy young participant of same gender. The T-score bone density categories were: normal (score \\>-1), osteopenia (score -2.5 to \\<=-1), and osteoporosis (score \\<= -2.5).", 'unitOfMeasure': 'T-Score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}, {'type': 'SECONDARY', 'title': 'Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'classes': [{'title': 'Lumbar Spine Z-Score: Baseline (n= 25)', 'categories': [{'measurements': [{'value': '-1.49', 'spread': '1.055', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 1 (n= 20)', 'categories': [{'measurements': [{'value': '-1.11', 'spread': '0.961', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 2 (n= 17)', 'categories': [{'measurements': [{'value': '-0.64', 'spread': '0.999', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 3 (n= 15)', 'categories': [{'measurements': [{'value': '-0.63', 'spread': '0.984', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 4 (n= 15)', 'categories': [{'measurements': [{'value': '-0.48', 'spread': '1.073', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 5 (n= 15)', 'categories': [{'measurements': [{'value': '-0.37', 'spread': '0.932', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 6 (n= 15)', 'categories': [{'measurements': [{'value': '-0.27', 'spread': '1.100', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 7 (n= 15)', 'categories': [{'measurements': [{'value': '-0.14', 'spread': '1.053', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 8 (n= 14)', 'categories': [{'measurements': [{'value': '-0.29', 'spread': '1.088', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '-0.05', 'spread': '0.885', 'groupId': 'OG000'}]}]}, {'title': 'Lumbar Spine Z-Score: End of Study (n= 6)', 'categories': [{'measurements': [{'value': '0.18', 'spread': '0.968', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Baseline (n= 23)', 'categories': [{'measurements': [{'value': '-0.17', 'spread': '0.783', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 1 (n= 19)', 'categories': [{'measurements': [{'value': '0.04', 'spread': '0.813', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 2 (n= 15)', 'categories': [{'measurements': [{'value': '0.25', 'spread': '0.787', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 3 (n= 13)', 'categories': [{'measurements': [{'value': '0.52', 'spread': '0.704', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 4 (n= 13)', 'categories': [{'measurements': [{'value': '0.48', 'spread': '0.773', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 5 (n= 13)', 'categories': [{'measurements': [{'value': '0.49', 'spread': '0.741', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 6 (n= 13)', 'categories': [{'measurements': [{'value': '0.57', 'spread': '0.665', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 7 (n= 13)', 'categories': [{'measurements': [{'value': '0.52', 'spread': '0.683', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 8 (n= 12)', 'categories': [{'measurements': [{'value': '0.64', 'spread': '0.693', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: Year 9 (n= 4)', 'categories': [{'measurements': [{'value': '0.38', 'spread': '0.793', 'groupId': 'OG000'}]}]}, {'title': 'Femur Z-Scores: End of Study (n= 6)', 'categories': [{'measurements': [{'value': '0.73', 'spread': '0.766', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)', 'description': 'Images of the lumbar spine and femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score \\>-2) and below normal (score \\<=-2).', 'unitOfMeasure': 'Z-Score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat. Here 'n' signifies number of participants with available data at specified time points."}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 milligram (mg) dose on Day 1 then eliglustat 50 mg twice daily (BID) from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 nanogram per milliliter \\[ng/mL\\] on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme), and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '26'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '19'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Undefined', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}]}], 'preAssignmentDetails': 'A total of 50 participants were screened of which 24 participants were screen failure. A total of 26 participants were enrolled in this study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Eliglustat', 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg BID from Day 2 to Day 19, and then either eliglustat 50 mg BID (if Genz-99067 \\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\] 5 ng/mL on Day 10) or eliglustat 100 mg BID (if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing the same treatment up to Year 9. Participant receiving 100 mg BID could be considered for a further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '34.47', 'spread': '12.960', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Gender', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '10', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Ashkenazi Jewish', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}, {'title': 'Non-Jewish Caucasian', 'categories': [{'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}, {'title': 'Hispanic', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Body Mass Index (BMI)', 'classes': [{'categories': [{'measurements': [{'value': '22.56', 'spread': '3.529', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'BMI was calculated as (\\[weight in kg\\] divided by \\[height in cm multiplied by 0.01\\]\\^2).', 'unitOfMeasure': 'kilogram per square meter (kg/m^2)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Weight', 'classes': [{'categories': [{'measurements': [{'value': '61.47', 'spread': '11.018', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kilogram (kg)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Height', 'classes': [{'categories': [{'measurements': [{'value': '165.12', 'spread': '9.747', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'centimeter (cm)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Hemoglobin', 'classes': [{'categories': [{'measurements': [{'value': '11.10', 'spread': '1.674', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'gram per deciliter (g/dL)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Platelet Count', 'classes': [{'categories': [{'measurements': [{'value': '66.423', 'spread': '20.1413', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': '10^9 cells per liter', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Spleen Volume', 'classes': [{'categories': [{'measurements': [{'value': '20.04', 'spread': '12.798', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Multiples of normal', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Full analysis set consists of all participants who signed informed consent and received at least one dose of eliglustat.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 26}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2006-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-12', 'dispFirstSubmitDate': '2011-12-16', 'completionDateStruct': {'date': '2015-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2016-12-22', 'studyFirstSubmitDate': '2006-07-27', 'dispFirstSubmitQcDate': '2011-12-16', 'resultsFirstSubmitDate': '2014-08-22', 'studyFirstSubmitQcDate': '2006-07-27', 'dispFirstPostDateStruct': {'date': '2011-12-22', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2017-02-15', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2014-08-22', 'studyFirstPostDateStruct': {'date': '2006-07-31', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-09-03', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2009-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants Demonstrating A Meaningful Clinical Response', 'timeFrame': 'Baseline, Year 1', 'description': 'A meaningful clinical response was defined as an improvement in at least 2 of the 3 main efficacy parameters: a) an increase in hemoglobin of greater than or equal to (\\>=) 0.5 gram/deciliter from baseline, b) an increase in platelets of \\>=15 percent (%) from baseline, c) reduction in total spleen volume of \\>= 15% from baseline. As hemoglobin, platelets, total spleen volume were abnormal at baseline, within each participant, only those parameters were used in the evaluation of meaningful clinical response which were abnormal at baseline.'}], 'secondaryOutcomes': [{'measure': 'Percent Change From Baseline in Spleen Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Percent change in spleen volume = (\\[spleen volume at specified time points minus spleen volume at baseline\\] divided by \\[spleen volume at baseline\\]) multiplied by 100, where all volumes are in multiples of normal.'}, {'measure': 'Percent Change From Baseline in Liver Volume at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Percent change in liver volume = (\\[liver volume at specified time points minus liver volume at baseline\\] divided by \\[liver volume at baseline\\]) multiplied by 100, where all volumes are in multiples of normal.'}, {'measure': 'Absolute Change From Baseline in Hemoglobin at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Absolute change = hemoglobin level at specified time points minus hemoglobin level at baseline.'}, {'measure': 'Percent Change From Baseline in Platelet Count at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Percent change in platelet count = (\\[platelet count at specified time points minus platelet count at baseline\\] divided by \\[platelet count at baseline\\]) multiplied by 100.'}, {'measure': 'Percent Change From Baseline in Biomarker (Angiotensin Converting Enzyme) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)'}, {'measure': 'Percent Change From Baseline in Biomarker (Tartrate-Resistant Acid Phosphatase [TRAP]) Level at Year 1 and Year 2', 'timeFrame': 'Baseline, Year 1, Year 2'}, {'measure': 'Percent Change From Baseline in Biomarker Chemokine Ligand 18 (CCL18) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and End of Study (Up to Year 9)'}, {'measure': 'Percent Change From Baseline in Biomarker (Chitotriosidase) Level at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)'}, {'measure': 'Change From Baseline in 36-Item Short Form (SF-36) Health Survey Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and Year 9 at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': "The SF-36 questionnaire, version 2, investigates the participant's health-related quality of life (HRQL). It is a 36-item questionnaire measuring 8 domains (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life. Two summary scale scores were computed from the 8 domain scores: the Physical Component Summary and the Mental Component Summary. Score range for both summary scale ranges from 0 (worst) to 100 (best), with higher scores reflecting best health-related quality of life."}, {'measure': 'Change From Baseline in Fatigue Severity Scale (FSS) Scores at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'The FSS is an instrument consisting of 9 self-administered questions that measures the impact of severity of fatigue symptoms on everyday functioning, based on the recall over the past week. Score range for each question ranges from 1 (minimum) to 7 (maximum), where higher score indicates greater severity. FSS total score was calculated by averaging the results of all questions. Total FSS score ranges from 9 (minimum) to 63 (maximum), where higher scores indicates greater severity.'}, {'measure': 'Number of Participants With Bone Pain Levels During the Past 4 Weeks at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain and moderate bone pain. In this outcome, number of participants with different levels of bone pain at specified time points were reported.'}, {'measure': 'Number of Participants With Mobility Status (MS) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Mobillity, i.e. ability to walk was assessed as a part of Gaucher disease assessment in participants.In this outcome, number of participants with their different mobility status (unrestricted mobility, walks with difficulty) at specified time points were reported.'}, {'measure': 'Number of Participants With No Bone Crisis at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (Up to Year 9)', 'description': 'Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes debilitation lasting several days or longer and requires treatment with immobilization, hydration, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, and 2= 2 bone crises during the assessment period. In this outcome, number of participants with 0= no bone crises levels at specified time points were reported.'}, {'measure': 'Bone Marrow Infiltration: Number of Participants With Improvement From Baseline in Dark Marrow at Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (EOS)', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8 and at End of Study (up to Year 9)', 'description': 'Bone marrow infiltration assessments were designed to evaluate improvements in dark marrow using MRI. Each MRI assessment was performed for both femurs and consisted of reviewing 6 different zones (the femoral head, greater trochanter, intertrochanteric region, shaft, distal metaphysis, and condyles). MRI images recorded dark marrow for each zone as either present or not present at baseline. In this outcome, number of participants (for whom dark marrow was present at baseline) with improvement from baseline in dark marrow at each specified time point were reported.'}, {'measure': 'Lumbar Spine and Femur T-Scores for Bone Mineral Density (BMD) at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)', 'description': "Images of the lumbar spine and femur were obtained by dual energy X-ray absorptiometry (DXA) to determine T-score for each bone area and total bone mineral density. T-scores compares participant's bone density with that of healthy young participant of same gender. The T-score bone density categories were: normal (score \\>-1), osteopenia (score -2.5 to \\<=-1), and osteoporosis (score \\<= -2.5)."}, {'measure': 'Lumbar Spine and Femur Z-Scores for BMD at Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study', 'timeFrame': 'Baseline, Year 1, Year 2, Year 3, Year 4, Year 5, Year 6, Year 7, Year 8, Year 9 and at End of Study (up to Year 9)', 'description': 'Images of the lumbar spine and femur were obtained by DXA to determine Z-score for each bone area and total bone mineral density. The Z-score bone density categories are: normal (score \\>-2) and below normal (score \\<=-2).'}]}, 'conditionsModule': {'keywords': ['Type 1 Gaucher Disease', 'Glucocerebrosidase Deficiency Disease'], 'conditions': ['Gaucher Disease, Type 1', 'Cerebroside Lipidosis Syndrome', 'Glucocerebrosidase Deficiency Disease', 'Glucosylceramide Beta-Glucosidase Deficiency Disease', 'Gaucher Disease, Non-Neuronopathic Form']}, 'referencesModule': {'references': [{'pmid': '17509920', 'type': 'BACKGROUND', 'citation': 'McEachern KA, Fung J, Komarnitsky S, Siegel CS, Chuang WL, Hutto E, Shayman JA, Grabowski GA, Aerts JM, Cheng SH, Copeland DP, Marshall J. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol Genet Metab. 2007 Jul;91(3):259-67. doi: 10.1016/j.ymgme.2007.04.001. Epub 2007 May 16.'}, {'pmid': '20864621', 'type': 'BACKGROUND', 'citation': 'Peterschmitt MJ, Burke A, Blankstein L, Smith SE, Puga AC, Kramer WG, Harris JA, Mathews D, Bonate PL. Safety, tolerability, and pharmacokinetics of eliglustat tartrate (Genz-112638) after single doses, multiple doses, and food in healthy volunteers. J Clin Pharmacol. 2011 May;51(5):695-705. doi: 10.1177/0091270010372387. Epub 2010 Sep 23.'}, {'pmid': '20439622', 'type': 'RESULT', 'citation': 'Lukina E, Watman N, Arreguin EA, Banikazemi M, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Rosenthal DI, Kaper M, Singh T, Puga AC, Bonate PL, Peterschmitt MJ. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Blood. 2010 Aug 12;116(6):893-9. doi: 10.1182/blood-2010-03-273151. Epub 2010 May 3.'}, {'pmid': '20713962', 'type': 'RESULT', 'citation': 'Lukina E, Watman N, Arreguin EA, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Kamath RS, Rosenthal DI, Kaper M, Singh T, Puga AC, Peterschmitt MJ. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood. 2010 Nov 18;116(20):4095-8. doi: 10.1182/blood-2010-06-293902. Epub 2010 Aug 16.'}, {'pmid': '24816856', 'type': 'RESULT', 'citation': 'Kamath RS, Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Aguzzi R, Puga AC, Norfleet AM, Peterschmitt MJ, Rosenthal DI. Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat. Skeletal Radiol. 2014 Oct;43(10):1353-60. doi: 10.1007/s00256-014-1891-9. Epub 2014 May 10.'}, {'pmid': '24835462', 'type': 'RESULT', 'citation': 'Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Angell J, Ross L, Puga AC, Peterschmitt JM. Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment. Blood Cells Mol Dis. 2014 Dec;53(4):274-6. doi: 10.1016/j.bcmd.2014.04.002. Epub 2014 May 15.'}, {'pmid': '36739645', 'type': 'DERIVED', 'citation': 'Peterschmitt MJ, Foster MC, Ji AJ, Zajdel MB, Cox GF. Plasma glucosylsphingosine correlations with baseline disease burden and response to eliglustat in two clinical trials of previously untreated adults with Gaucher disease type 1. Mol Genet Metab. 2023 Mar;138(3):107527. doi: 10.1016/j.ymgme.2023.107527. Epub 2023 Jan 25.'}]}, 'descriptionModule': {'briefSummary': 'Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid beta-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a substrate (lipid) called glucosylceramide and, to a lesser degree, glucosylsphingosine. In participants with Gaucher disease, the liver, spleen, bone marrow and brain show increases in lipid concentration, specifically in cells derived from the monocyte/macrophage system.\n\nEliglustat tartrate (Genz-112638) is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide. The primary objective of this study is to evaluate the efficacy, safety and pharmacokinetics (PK) of eliglustat tartrate, administered as an oral dose of either 50 milligram (mg) twice daily (BID) or 100 mg BID, to men and women with Gaucher disease Type 1 for 52 weeks.', 'detailedDescription': 'This study consists of several phases: screening (-28 to -1 days), dose adjustment/treatment (Day 1 \\[treatment baseline\\] to Day 30), initial steady-state treatment (post-Day 30 through Week 52 post-baseline), a treatment interruption period (Week 52 through approximately Week 54), long-term steady-state treatment (approximately Week 54 through study completion), and safety follow-up (30 to 37 days after a participant withdraws from or completes the study). The Primary Analysis Period is from baseline through Week 52. The Extension Period is from Week 52 through study completion (that is, participant withdrawal, the study is terminated, eliglustat tartrate becomes commercially available, or where applicable, specific regulatory requirements have been met).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* The participant had a diagnosis of Gaucher Type I disease and a documented deficiency of glucocerebrosidase activity by enzyme assay and was willing and able to provide written informed consent prior to initiating any study-related procedures;\n* The participant was 18 to 65 years old and weighed between 50 and 120 kilogram (kg) at enrollment;\n* The participant had the following symptoms of Gaucher disease identified within 28 days of enrollment (at screening);\n\n * Anemia - indicated by hemoglobin measurements taken during the screening phase (8 to 10 gram per deciliter (g/dL) if female, 8 to 11 g/dL if male);\n * Thrombocytopenia - indicated by platelet count measurements taken during the screening phase (60000 to 100000 per cubic millimeter);\n * Splenomegaly, as indicated by magnetic resonance imaging (MRI) or spiral computed tomography (CT) (\\>= 10 multiples of normal);\n* Female participants of child-bearing potential must had a documented negative serum pregnancy test prior to dosing. Female participants agreed to use a reliable method of birth control throughout duration of trial.\n\nExclusion Criteria:\n\n* Participant had a partial or total splenectomy or infarcted areas of the spleen;\n* Participant had documented prior bleeding varices or liver infarction;\n* Participant received miglustat within 12 months prior to study enrollment;\n* The participant had received an investigational product within 30 days prior to study enrollment;\n* Participant had neurologic or pulmonary involvement;\n* Participant had new pathological bone involvement or bone crisis in the 12 months prior to enrollment;\n* Participant was transfusion-dependent;\n* Participant had a documented etiology of anemia due to causes other than Gaucher disease;\n* The participant had cardiac functional and/or anatomical abnormalities, a history of cancer or tested positive for human immunodeficiency virus (HIV) antibody or Hepatitis;\n* Participant had a clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic, or psychiatric disease, other medical conditions, or serious intercurrent illnesses that, in the opinion of the Investigator, might preclude participation in the study.'}, 'identificationModule': {'nctId': 'NCT00358150', 'briefTitle': 'A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sanofi'}, 'officialTitle': 'A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients', 'orgStudyIdInfo': {'id': 'GZGD00304'}, 'secondaryIdInfos': [{'id': '2005-004732-42', 'type': 'EUDRACT_NUMBER'}, {'id': 'DRI12816', 'type': 'OTHER', 'domain': 'Sanofi'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Eliglustat tartrate', 'interventionNames': ['Drug: Eliglustat tartrate']}], 'interventions': [{'name': 'Eliglustat tartrate', 'type': 'DRUG', 'otherNames': ['Genz-112638'], 'description': 'Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg twice daily (BID) from Day 2 to Day 19, then either eliglustat 50 mg BID (if Genz-99067\\[active moiety of eliglustat in plasma\\] trough plasma concentration was greater than or equal to \\[\\>=\\]5 nanogram per milliliter \\[ng/mL\\] on Day 10) or eliglustat 100 mg BID(if Genz-99067 trough plasma concentration was less than \\[\\<\\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing same treatment through study completion (Year 9). Participant receiving 100 mg BID could be considered for further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).', 'armGroupLabels': ['Eliglustat tartrate']}]}, 'contactsLocationsModule': {'locations': [{'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'New York University', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'city': 'New York', 'state': 'New York', 'country': 'United States', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Aprillus Asistencia e Investigación', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Hospital de Oncologia Maria Curie', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Hospital Ramos Mejia', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'IMAI', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Instituto Argentino de Diagnostico y Tratamiento (IADT)', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Haifa', 'country': 'Israel', 'facility': 'Rambam Medical Center', 'geoPoint': {'lat': 32.81303, 'lon': 34.99928}}, {'city': 'Haifa', 'country': 'Israel', 'geoPoint': {'lat': 32.81303, 'lon': 34.99928}}, {'city': 'Jerusalem', 'country': 'Israel', 'facility': "Sha'are Zedek Medical Centre", 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'city': 'Jerusalem', 'country': 'Israel', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'city': 'Milan', 'country': 'Italy', 'facility': 'Universita degli Studi di Milano', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'city': 'D.f.', 'country': 'Mexico', 'facility': 'Instituto Mexicano del Seguro Social'}, {'city': 'Mexico City', 'country': 'Mexico', 'geoPoint': {'lat': 19.42847, 'lon': -99.12766}}, {'city': 'Moscow', 'country': 'Russia', 'facility': 'Hematology Research Center of Ministry of Healthcare of the Russian Federation', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'city': 'Moscow', 'country': 'Russia', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}], 'overallOfficials': [{'name': 'Medical Monitor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Genzyme, a Sanofi Company'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genzyme, a Sanofi Company', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}