21 completed. 21 Phase 3, 2 Phase 2.
By phase and status
Follow Gaucher Disease: new trials by email Literature Review of Gaucher Disease trials Research Report on Gaucher Disease
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All 21 trials
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix .
Trial Phase Status Sponsor Enrollment Start Updated
Role of Vitamin E in Gaucher Disease Patients Vitamin E - Gaucher Disease Phase 3 Completed 40 2023-03-03 2024-01-18 A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease Velaglucerase Alfa - Gaucher Disease Phase 3 Completed 20 2023-01-03 2025-06-26 Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 Eliglustat GZ385660, Imiglucerase GZ437843 - Gaucher's Disease Type I, Gaucher's Disease Type III Phase 3 Completed 57 2018-04-11 2026-06-16 Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies Eliglustat, GZ385660 - Gaucher Disease Phase 3 Completed 31 2015-10-27 2022-07-15 Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease velaglucerase alfa - Gaucher Disease Phase 3 Completed 5 2013-03-13 2021-06-14 Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease velaglucerase alfa - Gaucher Disease Phase 3 Completed 6 2012-03-02 2021-06-28 A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease Taliglucerase alfa - Gaucher Disease Phase 3 Completed 15 2011-09-01 2018-09-07 Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease ISU302 - Gaucher Disease, Type 1 Phase 3 Completed 8 2011-09-01 2017-07-12 A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease Taliglucerase alfa - Gaucher Disease Phase 3 Completed 19 2011-08-01 2023-04-19 A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE) Eliglustat tartrate - Gaucher Disease Phase 3 Completed 170 2010-06-01 2017-02-06 A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) Eliglustat tartrate, Placebo - Gaucher Disease, Type 1 Phase 3 Completed 40 2009-11-01 2017-03-03 A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) Eliglustat tartrate, Imiglucerase - Gaucher Disease, Type 1 Phase 3 Completed 160 2009-09-01 2016-11-25 Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase Taliglucerase alfa - Gaucher Disease Phase 3 Completed 31 2008-12-01 2018-10-04 Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial Taliglucerase alfa - Gaucher Disease Phase 3 Completed 45 2008-06-01 2018-10-04 An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease VPRIV® - Gaucher Disease, Type 1 Phase 3 Completed 95 2008-03-13 2021-06-10 Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease velaglucerase alfa, imiglucerase - Gaucher Disease, Type 1 Phase 3 Completed 34 2008-01-29 2021-06-08 A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease Plant cell expressed recombinant glucocerebrosidase (prGCD) - Gaucher Disease Phase 3 Completed 32 2007-08-01 2018-10-04 Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase GA-GCB (velaglucerase alfa) - Gaucher Disease Phase 2 / Phase 3 Completed 40 2007-07-25 2021-06-10 A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease VPRIV ®, - Gaucher Disease, Type 1 Phase 3 Completed 25 2007-02-15 2021-06-29 Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease Miglustat - Gaucher Disease Type 1 Phase 3 Completed 42 2006-02-01 2025-02-04
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Narrower conditions Adrenoleukodystrophy (1) · Fucosidosis (1) · Gangliosidosis, GM1 (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Lysosomal Storage Diseases (1) · Neuronal Ceroid-Lipofuscinoses (1) · Niemann-Pick Diseases (1) · Sandhoff Disease (1) · Tay-Sachs Disease (1) · Wolman Disease (1)
Broader conditions Brain Diseases (21) · Brain Diseases, Metabolic (21) · Brain Diseases, Metabolic, Inborn (21) · Central Nervous System Diseases (21) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (21) · Genetic Diseases, Inborn (21) · Lipid Metabolism Disorders (21) · Lipid Metabolism, Inborn Errors (21) · Lipidoses (21) · Lysosomal Storage Diseases, Nervous System (21) · Metabolic Diseases (21) · Metabolism, Inborn Errors (21) · Nervous System Diseases (21) · Nutritional and Metabolic Diseases (21) · Sphingolipidoses (21)
Interventions Glucosylceramidase (7) · eliglustat (5) · taliglucerase alfa (5) · imiglucerase (4) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Stem Cell Transplantation (1) · Vitamin E (1) · miglustat (1)
Sponsors Shire (6) · Pfizer (5) · Genzyme, a Sanofi Company (4) · Actelion (1) · Ain Shams University (1) · ISU Abxis Co., Ltd. (1) · Masonic Cancer Center, University of Minnesota (1) · Sanofi (1) · Takeda (1)
Countries United States (10) · Israel (9) · United Kingdom (9) · Canada (8) · Russia (8) · Spain (8) · Argentina (5) · Tunisia (5) · Australia (4) · India (4) · Japan (4) · Paraguay (4) · France (3) · Italy (3) · South Africa (3)
Top investigators Amel El Beshlawy, Prof. (1) · Amira A Adly, Phd (1) · Paul Orchard, MD (1) · Timothy Cox, Prof (1)
Results publications PMID 27839979 , PMID 26053270 , PMID 18729283 , PMID 21586746 , PMID 21900191 , PMID 23270487 , PMID 23339116 , PMID 24950666 , PMID 25688781 , PMID 25801797 , PMID 25819691 , PMID 26043810 , PMID 27102949 , PMID 27174694 , PMID 27241455 , PMID 27499018 , PMID 27839981 , PMID 28167660 , PMID 29358012 , PMID 34161616 , PMID 36739645 , PMID 39183538 , PMID 39774103
NIH funding 2008 (3) , 2009 (1) , 2016 (1)