{"disease":{"id":"gaucher-s-disease","name":"gaucher s disease"},"drugs":{"marketed":[{"drug_id":"glucocerebrosidase","indication_name":"Chronic non-neuropathic Gaucher's disease","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":null,"generic_name":"glucocerebrosidase","company_name":"National Center for Research Resources (NCRR)","drug_phase":"phase_2","molecular_target":"","drug_class":"","quality_score":18,"revenue":null,"mechanism":null},{"drug_id":"alglucerase","indication_name":"Chronic non-neuropathic Gaucher's disease","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Ceredase","generic_name":"ALGLUCERASE","company_name":"Sanofi","drug_phase":"marketed","molecular_target":"","drug_class":"Hydrolytic Lysosomal Glucocerebroside-specific Enzyme","quality_score":25,"revenue":null,"mechanism":""},{"drug_id":"velaglucerase-alfa","indication_name":"Chronic non-neuropathic Gaucher's disease","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Vpriv","generic_name":"VELAGLUCERASE ALFA","company_name":"Shire Human Genetic","drug_phase":"marketed","molecular_target":"","drug_class":"Hydrolytic Lysosomal Glucocerebroside-specific Enzyme [EPC]","quality_score":50,"revenue":null,"mechanism":"VELAGLUCERASE ALFA replaces the deficient enzyme glucocerebrosidase in patients with Gaucher's disease."}],"pipeline":[],"offLabel":[],"totalMarketed":3,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT05368038","title":"ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":100000,"lead_sponsor_name":"Albert Einstein College of Medicine","has_results":false},{"nct_id":"NCT00358943","title":"International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry","phase":"","overall_status":"RECRUITING","enrollment_count":12000,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":false},{"nct_id":"NCT00001215","title":"Genetic Studies of Lysosomal Storage Disorders","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":1000,"lead_sponsor_name":"National Human Genome Research Institute (NHGRI)","has_results":false},{"nct_id":"NCT06539169","title":"FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","phase":"","overall_status":"RECRUITING","enrollment_count":1000,"lead_sponsor_name":"xCures","has_results":false},{"nct_id":"NCT06573723","title":"Institutional Registry of Rare Diseases","phase":"","overall_status":"RECRUITING","enrollment_count":380,"lead_sponsor_name":"Hospital Italiano de Buenos Aires","has_results":false},{"nct_id":"NCT04388969","title":"World Data on Ambroxol for Patients With GD and GBA Related PD","phase":"","overall_status":"UNKNOWN","enrollment_count":300,"lead_sponsor_name":"Shaare Zedek Medical Center","has_results":false},{"nct_id":"NCT03240653","title":"Gaucherite - A Study to Stratify Gaucher Disease","phase":"","overall_status":"RECRUITING","enrollment_count":250,"lead_sponsor_name":"Cambridge University Hospitals NHS Foundation Trust","has_results":false},{"nct_id":"NCT05619900","title":"Registry of Patients Diagnosed With Lysosomal Storage Diseases","phase":"","overall_status":"RECRUITING","enrollment_count":250,"lead_sponsor_name":"University of California, San Francisco","has_results":false},{"nct_id":"NCT04470713","title":"Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2","phase":"","overall_status":"COMPLETED","enrollment_count":226,"lead_sponsor_name":"Idorsia Pharmaceuticals Ltd.","has_results":false},{"nct_id":"NCT04758130","title":"Getting Global Rare Disease Insights Through Technology Study","phase":"NA","overall_status":"UNKNOWN","enrollment_count":150,"lead_sponsor_name":"M.A.G.I.C. Clinic LTD","has_results":false},{"nct_id":"NCT05641103","title":"PREDIGA 2: Spanish Acronym of \"Educational and Diagnostic Project for Gaucher and ASMD\"","phase":"","overall_status":"COMPLETED","enrollment_count":122,"lead_sponsor_name":"Fundación Española de Hematología y Hemoterapía","has_results":false},{"nct_id":"NCT00001289","title":"Effects of Enzyme Replacement in Gaucher's Disease","phase":"","overall_status":"COMPLETED","enrollment_count":70,"lead_sponsor_name":"National Institute of Neurological Disorders and Stroke (NINDS)","has_results":false},{"nct_id":"NCT03485677","title":"Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":57,"lead_sponsor_name":"Sanofi","has_results":false},{"nct_id":"NCT05222906","title":"Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":43,"lead_sponsor_name":"Sanofi","has_results":false},{"nct_id":"NCT05447494","title":"Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease","phase":"PHASE1, PHASE2","overall_status":"UNKNOWN","enrollment_count":40,"lead_sponsor_name":"CANbridge (Suzhou) Bio-pharma Co., Ltd.","has_results":false},{"nct_id":"NCT03746587","title":"Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3","phase":"PHASE2","overall_status":"TERMINATED","enrollment_count":39,"lead_sponsor_name":"ZevraDenmark","has_results":false},{"nct_id":"NCT00433147","title":"A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"Amicus Therapeutics","has_results":true},{"nct_id":"NCT06116071","title":"Biomarkers Related to Bone in Pediatric Gaucher Disease","phase":"","overall_status":"UNKNOWN","enrollment_count":20,"lead_sponsor_name":"Lysosomal and Rare Disorders Research and Treatment Center, Inc.","has_results":false},{"nct_id":"NCT00446550","title":"A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":19,"lead_sponsor_name":"Amicus Therapeutics","has_results":true},{"nct_id":"NCT06188325","title":"A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":18,"lead_sponsor_name":"Sanofi","has_results":false},{"nct_id":"NCT05487599","title":"A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)","phase":"PHASE1, PHASE2","overall_status":"RECRUITING","enrollment_count":15,"lead_sponsor_name":"Prevail Therapeutics","has_results":false},{"nct_id":"NCT02843035","title":"Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension","phase":"PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":12,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":false},{"nct_id":"NCT04656600","title":"Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ","phase":"PHASE4","overall_status":"COMPLETED","enrollment_count":12,"lead_sponsor_name":"Sanofi","has_results":false},{"nct_id":"NCT04532047","title":"PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)","phase":"PHASE1","overall_status":"RECRUITING","enrollment_count":10,"lead_sponsor_name":"University of California, San Francisco","has_results":false},{"nct_id":"NCT06143904","title":"A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":10,"lead_sponsor_name":"Sanofi","has_results":false},{"nct_id":"NCT00813865","title":"A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":8,"lead_sponsor_name":"Amicus Therapeutics","has_results":true},{"nct_id":"NCT06193304","title":"A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":6,"lead_sponsor_name":"Sanofi","has_results":false},{"nct_id":"NCT04050137","title":"Therapeutic Exercise to Treat Neuropathic Pain","phase":"NA","overall_status":"COMPLETED","enrollment_count":5,"lead_sponsor_name":"Universidad de Zaragoza","has_results":false},{"nct_id":"NCT04189601","title":"Complement Activation in the Lysosomal Storage Disorders","phase":"","overall_status":"WITHDRAWN","enrollment_count":0,"lead_sponsor_name":"Melbourne Health","has_results":false}],"total":29},"guidelines":[],"source":"Drug Landscape verified database"}