Ignite Creation Date:
2025-12-25 @ 3:56 AM
Ignite Modification Date:
2025-12-26 @ 2:46 AM
Study NCT ID:
NCT00891202
Status:
COMPLETED
Last Update Posted:
2017-03-03
First Post:
2009-04-30
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Chile', 'Georgia', 'Jordan', 'Netherlands', 'Saudi Arabia']}, '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}}, 'adverseEventsModule': {'timeFrame': 'Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).', 'description': "Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).", 'eventGroups': [{'id': 'EG000', 'title': 'Eliglustat', 'description': 'PAP: Eliglustat tartrate capsule 50 mg orally on Day 1 followed by eliglustat tartrate 50 mg capsule BID from Day 2 to Week 4, then either eliglustat tartrate 50 mg capsule BID(participants with Genz-99067 trough plasma concentration\\>=5 ng/mL) or eliglustat tartrate 100 mg capsule BID(participants with Genz-99067 trough plasma concentration\\<5 ng/mL), up to Week 39. PK assessment at Week 2 used for dose adjustment after Week 4. LTTP: Participants of the eliglustat arm in PAP who completed PAP were included in LTTP \\& received eliglustat tartrate capsule 50 mg BID orally from Day 1(post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 \\& Week 47 were based on Genz-99067 trough plasma concentrations(if trough plasma concentration\\<5 ng/mL: next higher dose administered;if\\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.', 'otherNumAtRisk': 20, 'otherNumAffected': 17, 'seriousNumAtRisk': 20, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'Placebo', 'description': 'PAP: Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. LTTP: Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline for LTTP. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.', 'otherNumAtRisk': 20, 'otherNumAffected': 14, 'seriousNumAtRisk': 20, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Iron deficiency anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 3}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Eye irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gastritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Hordeolum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Thermal burn', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Blood creatine phosphokinase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Bone density decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Bone pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Joint stiffness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 7}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Dysmenorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Nasal obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Acne', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Skin lesion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}], 'seriousEvents': [{'term': 'Atrioventricular block', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Atrioventricular block second degree', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Ventricular tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Biliary colic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}, {'term': 'Osteoarthritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 20, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 20, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo', 'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'PAP: Eliglustat', 'description': 'Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.'}, {'id': 'OG001', 'title': 'PAP: Placebo', 'description': 'Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.'}], 'classes': [{'categories': [{'measurements': [{'value': '-27.77', 'spread': '2.37', 'groupId': 'OG000'}, {'value': '2.26', 'spread': '2.37', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Least Squares Mean Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-30.03', 'ciLowerLimit': '-36.82', 'ciUpperLimit': '-23.24', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '3.35', 'groupDescription': 'Analysis was performed using analysis of covariance (ANCOVA) model fitted with treatment and baseline spleen severity (low spleen severity: spleen volume less than or equal to \\[\\<=\\] 20 multiples of normal spleen volume, high spleen severity: spleen volume greater than \\[\\>\\] 20 multiples of normal spleen volume).', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'PAP Baseline (Day 1), Week 39', 'description': 'Percent change in spleen volume = (\\[spleen volume at Week 39 minus spleen volume at baseline\\] divided by \\[spleen volume at baseline\\]) multiplied by 100, where all volumes are in MN.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).'}, {'type': 'SECONDARY', 'title': 'PAP: Hemoglobin Level', 'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'PAP: Eliglustat', 'description': 'Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.'}, {'id': 'OG001', 'title': 'PAP: Placebo', 'description': 'Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.'}], 'classes': [{'categories': [{'measurements': [{'value': '12.05', 'spread': '1.816', 'groupId': 'OG000'}, {'value': '12.75', 'spread': '1.629', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PAP Baseline (Day 1)', 'unitOfMeasure': 'gram per deciliter (g/dL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).'}, {'type': 'SECONDARY', 'title': 'PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39', 'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'PAP: Eliglustat', 'description': 'Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.'}, {'id': 'OG001', 'title': 'PAP: Placebo', 'description': 'Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.69', 'spread': '0.23', 'groupId': 'OG000'}, {'value': '-0.54', 'spread': '0.23', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'PAP Baseline (Day 1), Week 39', 'description': 'Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline.', 'unitOfMeasure': 'g/dL', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).'}, {'type': 'SECONDARY', 'title': 'PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39', 'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'PAP: Eliglustat', 'description': 'Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.'}, {'id': 'OG001', 'title': 'PAP: Placebo', 'description': 'Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.'}], 'classes': [{'categories': [{'measurements': [{'value': '-5.20', 'spread': '1.64', 'groupId': 'OG000'}, {'value': '1.44', 'spread': '1.64', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'PAP Baseline (Day 1), Week 39', 'description': 'Percent change in liver volume = (\\[liver volume at Week 39 minus liver volume at baseline\\] divided by \\[liver volume at baseline\\]) multiplied by 100, where all volumes are in MN.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).'}, {'type': 'SECONDARY', 'title': 'PAP: Percent Change From Baseline in Platelet Counts at Week 39', 'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'PAP: Eliglustat', 'description': 'Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.'}, {'id': 'OG001', 'title': 'PAP: Placebo', 'description': 'Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.'}], 'classes': [{'categories': [{'measurements': [{'value': '32.00', 'spread': '5.95', 'groupId': 'OG000'}, {'value': '-9.06', 'spread': '5.95', 'groupId': 'OG001'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'PAP Baseline (Day 1), Week 39', 'description': 'Percent change in platelet count = (\\[platelet count at Week 39 minus platelet count at baseline\\] divided by \\[platelet count at baseline\\]) multiplied by 100.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).'}, {'type': 'SECONDARY', 'title': 'LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LTTP: Eliglustat (Originally on Eliglustat)', 'description': 'Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}, {'id': 'OG001', 'title': 'LTTP: Eliglustat (Originally on Placebo)', 'description': 'Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}], 'classes': [{'categories': [{'measurements': [{'value': '-66.9', 'spread': '8.45', 'groupId': 'OG000'}, {'value': '-64.0', 'spread': '6.43', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234', 'description': 'Percent change in spleen volume = (\\[spleen volume at Week 234 minus spleen volume at baseline\\] divided by \\[spleen volume at baseline\\]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent-to-treat (ITT) population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed= participants evaluable for this outcome measure and had available data for baseline and Week 234 spleen volume assessment.'}, {'type': 'SECONDARY', 'title': 'LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LTTP: Eliglustat (Originally on Eliglustat)', 'description': 'Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}, {'id': 'OG001', 'title': 'LTTP: Eliglustat (Originally on Placebo)', 'description': 'Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.1', 'spread': '0.65', 'groupId': 'OG000'}, {'value': '1.9', 'spread': '1.88', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234', 'description': 'Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.', 'unitOfMeasure': 'g/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 hemoglobin level assessment.'}, {'type': 'SECONDARY', 'title': 'LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LTTP: Eliglustat (Originally on Eliglustat)', 'description': 'Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}, {'id': 'OG001', 'title': 'LTTP: Eliglustat (Originally on Placebo)', 'description': 'Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}], 'classes': [{'categories': [{'measurements': [{'value': '-24.3', 'spread': '11.21', 'groupId': 'OG000'}, {'value': '-22.4', 'spread': '10.77', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234', 'description': 'Percent change in liver volume = (\\[liver volume at Week 234 minus liver volume at baseline\\] divided by \\[liver volume at baseline\\]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 liver volume assessment.'}, {'type': 'SECONDARY', 'title': 'LTTP: Percent Change From Baseline in Platelet Counts at Week 234', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'LTTP: Eliglustat (Originally on Eliglustat)', 'description': 'Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}, {'id': 'OG001', 'title': 'LTTP: Eliglustat (Originally on Placebo)', 'description': 'Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}], 'classes': [{'categories': [{'measurements': [{'value': '77.3', 'spread': '28.17', 'groupId': 'OG000'}, {'value': '100.1', 'spread': '80.69', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234', 'description': 'Percent change in platelet count = (\\[platelet count at Week 234 minus platelet count at baseline\\] divided by \\[platelet count at baseline\\]) multiplied by 100. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 platelet count assessment.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'PAP: Eliglustat', 'description': 'Eliglustat tartrate capsule as a single 50 milligram (mg) dose on Day 1 followed by eliglustat tartrate 50 mg capsule twice daily (BID) from Day 2 to Week 4, and then either eliglustat tartrate 50 mg capsule BID (in participants who had a Genz-99067 \\[active moiety of eliglustat tartrate in plasma\\] trough plasma concentration greater than or equal to \\[\\>=\\] 5 nanogram per milliliter \\[ng/mL\\]) or eliglustat tartrate 100 mg capsule BID (in participants who had a Genz-99067 trough plasma concentration less than \\[\\<\\] 5 ng/mL), up to Week 39. The pharmacokinetic (PK) assessment at Week 2 was used for dose adjustment after Week 4.'}, {'id': 'FG001', 'title': 'PAP: Placebo', 'description': 'Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.'}, {'id': 'FG002', 'title': 'LTTP: Eliglustat (Originally on Eliglustat)', 'description': 'Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}, {'id': 'FG003', 'title': 'LTTP: Eliglustat (Originally on Placebo)', 'description': 'Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.'}], 'periods': [{'title': 'PAP (Up To Week 39)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '20'}, {'groupId': 'FG001', 'numSubjects': '20'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '19'}, {'groupId': 'FG001', 'numSubjects': '20'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}, {'title': 'LTTP (Post-Week 39 up to Week 312)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '19'}, {'groupId': 'FG003', 'numSubjects': '20'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '12'}, {'groupId': 'FG003', 'numSubjects': '15'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '7'}, {'groupId': 'FG003', 'numSubjects': '5'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '4'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Pregnancy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Transitioned to commercial eliglustat', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '2'}, {'groupId': 'FG003', 'numSubjects': '5'}]}]}], 'recruitmentDetails': 'A total of 72 participants were screened between 5 November 2009 and 29 July 2011, of which 32 participants were screen failure. Overall 40 participants were enrolled and the study was conducted across 18 centers in 12 countries.', 'preAssignmentDetails': 'The 40 participants who met inclusion criteria received placebo or Genz-112638 (eliglustat tartrate) during 39 weeks primary analysis period (PAP). After Week 39 of the PAP, all participants who remained in the study received eliglustat tartrate in the long-term treatment period (LTTP) for up to Week 312.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '20', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '40', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'PAP: Eliglustat', 'description': 'Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.'}, {'id': 'BG001', 'title': 'PAP: Placebo', 'description': 'Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '31.6', 'spread': '11.55', 'groupId': 'BG000'}, {'value': '32.1', 'spread': '11.26', 'groupId': 'BG001'}, {'value': '31.8', 'spread': '11.26', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Gender', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '20', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Race: White', 'categories': [{'measurements': [{'value': '19', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '39', 'groupId': 'BG002'}]}]}, {'title': 'Race: Asian', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Ethnicity: Not Hispanic or Latino', 'categories': [{'measurements': [{'value': '18', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '38', 'groupId': 'BG002'}]}]}, {'title': 'Ethnicity: Hispanic or Latino', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Body Mass Index (BMI)', 'classes': [{'categories': [{'measurements': [{'value': '23.3', 'spread': '2.74', 'groupId': 'BG000'}, {'value': '23.4', 'spread': '3.54', 'groupId': 'BG001'}, {'value': '23.4', 'spread': '3.13', 'groupId': 'BG002'}]}]}], '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': '64.8', 'spread': '11.74', 'groupId': 'BG000'}, {'value': '68.6', 'spread': '17.17', 'groupId': 'BG001'}, {'value': '66.7', 'spread': '14.65', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kilogram (kg)', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Height', 'classes': [{'categories': [{'measurements': [{'value': '166.2', 'spread': '9.91', 'groupId': 'BG000'}, {'value': '170.0', 'spread': '12.02', 'groupId': 'BG001'}, {'value': '168.1', 'spread': '11.05', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'centimeter (cm)', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Full analysis set (FAS) for PAP: included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 40}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-01', 'dispFirstSubmitDate': '2012-10-12', 'completionDateStruct': {'date': '2016-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-01-17', 'studyFirstSubmitDate': '2009-04-30', 'dispFirstSubmitQcDate': '2012-10-12', 'resultsFirstSubmitDate': '2014-08-22', 'studyFirstSubmitQcDate': '2009-04-30', 'dispFirstPostDateStruct': {'date': '2012-10-16', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2017-03-03', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2014-08-22', 'studyFirstPostDateStruct': {'date': '2009-05-01', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-09-03', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo', 'timeFrame': 'PAP Baseline (Day 1), Week 39', 'description': 'Percent change in spleen volume = (\\[spleen volume at Week 39 minus spleen volume at baseline\\] divided by \\[spleen volume at baseline\\]) multiplied by 100, where all volumes are in MN.'}], 'secondaryOutcomes': [{'measure': 'PAP: Hemoglobin Level', 'timeFrame': 'PAP Baseline (Day 1)'}, {'measure': 'PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39', 'timeFrame': 'PAP Baseline (Day 1), Week 39', 'description': 'Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline.'}, {'measure': 'PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39', 'timeFrame': 'PAP Baseline (Day 1), Week 39', 'description': 'Percent change in liver volume = (\\[liver volume at Week 39 minus liver volume at baseline\\] divided by \\[liver volume at baseline\\]) multiplied by 100, where all volumes are in MN.'}, {'measure': 'PAP: Percent Change From Baseline in Platelet Counts at Week 39', 'timeFrame': 'PAP Baseline (Day 1), Week 39', 'description': 'Percent change in platelet count = (\\[platelet count at Week 39 minus platelet count at baseline\\] divided by \\[platelet count at baseline\\]) multiplied by 100.'}, {'measure': 'LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234', 'timeFrame': 'PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234', 'description': 'Percent change in spleen volume = (\\[spleen volume at Week 234 minus spleen volume at baseline\\] divided by \\[spleen volume at baseline\\]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.'}, {'measure': 'LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234', 'timeFrame': 'PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234', 'description': 'Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.'}, {'measure': 'LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234', 'timeFrame': 'PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234', 'description': 'Percent change in liver volume = (\\[liver volume at Week 234 minus liver volume at baseline\\] divided by \\[liver volume at baseline\\]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.'}, {'measure': 'LTTP: Percent Change From Baseline in Platelet Counts at Week 234', 'timeFrame': 'PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234', 'description': 'Percent change in platelet count = (\\[platelet count at Week 234 minus platelet count at baseline\\] divided by \\[platelet count at baseline\\]) multiplied by 100. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Gaucher,', 'beta-glucosidase,', 'acid ß-glucosidase,', 'glucocerebrosidase,', 'glucosylceramide,', 'D-glucosyl-N-acylsphingosine glucohydrolase,', 'substrate reduction therapy'], 'conditions': ['Gaucher Disease, Type 1']}, 'referencesModule': {'references': [{'pmid': '20439622', 'type': 'BACKGROUND', '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': 'BACKGROUND', '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': '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': '24816856', 'type': 'BACKGROUND', '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': 'BACKGROUND', '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': '25688781', 'type': 'RESULT', 'citation': 'Mistry PK, Lukina E, Ben Turkia H, Amato D, Baris H, Dasouki M, Ghosn M, Mehta A, Packman S, Pastores G, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Shankar S, Solano MH, Ross L, Angell J, Peterschmitt MJ. Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial. JAMA. 2015 Feb 17;313(7):695-706. doi: 10.1001/jama.2015.459.'}, {'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.'}, {'pmid': '34161616', 'type': 'DERIVED', 'citation': 'Mistry PK, Lukina E, Ben Turkia H, Shankar SP, Baris Feldman H, Ghosn M, Mehta A, Packman S, Lau H, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Foster MC, Gaemers SJM, Peterschmitt MJ. Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results. Am J Hematol. 2021 Sep 1;96(9):1156-1165. doi: 10.1002/ajh.26276. Epub 2021 Jul 11.'}]}, 'descriptionModule': {'briefSummary': 'This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.', 'detailedDescription': 'Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Type 1 Gaucher disease, the most common form accounts for greater than (\\>) 90% of cases and does not involve the central nervous system (CNS). Typical manifestations of Type 1 Gaucher disease include splenomegaly, hepatomegaly, thrombocytopenia, anemia, skeletal pathology and decreased quality of life. The disease manifestations are caused by the accumulations of glucosylceramide (storage material) in Gaucher cells which have infiltrated the spleen and liver as well as other tissue. Eliglustat tartrate is a small molecule developed as an oral therapy which acts to specifically inhibit production of this storage material in Gaucher cells.\n\nThis study was designed to determine the efficacy, safety, and pharmacokinetics (PK) of eliglustat tartrate in adult participants (\\>16 years) with Gaucher disease Type 1. The study consisted of 2 periods: The Double-Blind Primary Analysis Period (PAP \\[Day 1 to Week 39\\]) and the Long Term Treatment Period (LTTP/Open-Label Period (post-Week 39 \\[Day 1 of the Open-Label Period\\] through study completion).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* The participant (and/or their parent/legal guardian) was willing and able to provide signed informed consent prior to any study-related procedures to be performed;\n* The participant was at least 16 years old at the time of randomization;\n* The participant had a confirmed diagnosis of Gaucher disease Type 1;\n* Female participants of childbearing potential must had a documented negative pregnancy test prior to dosing. In addition all female participants of childbearing potential must use a medically accepted form of contraception throughout the study.\n\nExclusion Criteria:\n\n* The participant has had a partial or total splenectomy;\n* The participant had received pharmacological chaperones or miglustat within 6 months prior to randomization;\n* The participant had received enzyme replacement therapy within 9 months prior to randomization;\n* The participant had Type 2 or 3 Gaucher disease or was suspected of having Type 3 Gaucher disease;\n* The participant had any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal (GI), pulmonary, neurologic, endocrine, metabolic, (for example, hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illness that might confound the study results, or, on the opinion of the investigator, might preclude participation in the study;\n* The participant had tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen;\n* The participant had received an investigational product within 30 days prior to randomization;\n* The participant was pregnant or lactating.'}, 'identificationModule': {'nctId': 'NCT00891202', 'briefTitle': 'A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sanofi'}, 'officialTitle': 'A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE)', 'orgStudyIdInfo': {'id': 'GZGD02507'}, 'secondaryIdInfos': [{'id': '2008-005222-37', 'type': 'EUDRACT_NUMBER'}, {'id': 'EFC12813', 'type': 'OTHER', 'domain': 'Sanofi'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Active', 'description': 'Eliglustat', 'interventionNames': ['Drug: Eliglustat tartrate']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Eliglustat tartrate', 'type': 'DRUG', 'otherNames': ['Genz-112638'], 'description': 'PAP: Eliglustat tartrate (ET) capsule 50 mg orally on Day 1 followed by ET 50 mg capsule twice daily (BID) from Day 2 to Week 4, then either ET 50 mg capsule BID (participants with Genz-99067 \\[active moiety of ET in plasma\\] trough plasma concentration \\>=5 ng/mL) or ET 100 mg capsule BID (participants with Genz-99067 trough plasma concentration \\<5 ng/mL), up to Week 39. PK assessment at Week 2 used for dose adjustment after Week 4. LTTP: Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received ET capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by ET 50 mg or 100 mg capsule BID up to Week 47, then ET 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.', 'armGroupLabels': ['Active']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'PAP: Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. LTTP: Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline for LTTP. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \\<5 ng/mL: next higher dose administered; if \\>=5 ng/mL: same dose continued) at Week 41 \\& Week 45, respectively.', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94143', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'UCSF MS Center', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '06510', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale University School of Medicine', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '30033', 'city': 'Decatur', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University Medical Genetics', 'geoPoint': {'lat': 33.77483, 'lon': -84.29631}}, {'zip': '66160', 'city': 'Westwood', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center, Division of Hematology/Oncology, Dept. of Medicine', 'geoPoint': {'lat': 39.04056, 'lon': -94.6169}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'New York University School of Medicine, Neurology Department', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Mount Sinai School of Medicine', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '1431', 'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'University hospital "Alexandrovska" Sofia', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'zip': 'H3T 1E2', 'city': 'Montreal, Quebec', 'country': 'Canada', 'facility': 'Sir Mortimer B. Davis - Jewish General Hospital'}, {'zip': 'M5G 1X5', 'city': 'Toronto Ontario', 'country': 'Canada', 'facility': 'Mount Sinai Hospital and the Samuel Lunenfeld Research Institute'}, {'city': 'Bogotá', 'country': 'Colombia', 'facility': 'Hospital de San Jose', 'geoPoint': {'lat': 4.60971, 'lon': -74.08175}}, {'zip': '632004', 'city': 'Vellore', 'country': 'India', 'facility': 'Christian Medical College Hospital', 'geoPoint': {'lat': 12.9184, 'lon': 79.13255}}, {'zip': '49100', 'city': 'Petach Tikvah', 'country': 'Israel', 'facility': 'Rabin Medical Center, Beilinson Hospital'}, {'city': 'Beirut', 'country': 'Lebanon', 'facility': 'Hôtel-Dieu de France University Hospital', 'geoPoint': {'lat': 33.89332, 'lon': 35.50157}}, {'city': 'Monterrey, Nuevo Leon', 'country': 'Mexico', 'facility': 'OCA Hospital'}, {'zip': '125167', 'city': 'Moscow', 'country': 'Russia', 'facility': 'Hematology Research Center of Ministry of Healthcare of the Russian Federation', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'zip': '11000', 'city': 'Belgrade', 'country': 'Serbia', 'facility': 'Institut za endokrinologiju', 'geoPoint': {'lat': 44.80401, 'lon': 20.46513}}, {'zip': '1007', 'city': 'Tunis', 'state': 'TN', 'country': 'Tunisia', 'facility': 'Hopital La-Rabta', 'geoPoint': {'lat': 36.81897, 'lon': 10.16579}}, {'zip': 'NW3 2QG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Free Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Medical Monitor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Genzyme, a Sanofi Company'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genzyme, a Sanofi Company', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}