21 completed, 2 not yet recruiting, 2 withdrawn, 1 active not recruiting. 27 Phase 3, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 27 | Completed 21, Not yet recruiting 2, Withdrawn 2, Active not recruiting 1, Recruiting 1 |
| Phase 2 | 4 | Completed 2, Not yet recruiting 1, Withdrawn 1 |
Follow Gaucher Disease: new trials by email Literature Review of Gaucher Disease trials Research Report on Gaucher Disease
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 |
|---|---|---|---|---|---|---|
| Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders AZ-3102 - Niemann-Pick Disease Type C, GM2 Gangliosidosis | Phase 2 / Phase 3 | Not yet recruiting | Azafaros B.V. | 218 | 2026-12-10 | 2026-10-21 |
| Study of Velaglucerase Beta ERT in Children With Gaucher Disease Velaglucerase beta - Gaucher Disease, Gaucher Disease Type 1 | Phase 3 | Not yet recruiting | CANbridge Life Sciences Ltd. | 10 | 2026-09-16 | 2026-09-29 |
| A Gaucher Disease Gene Therapy Trial With FLT201 FLT201 - Gaucher Disease Type 1 | Phase 3 | Recruiting | Spur Therapeutics | 45 | 2026-04-07 | 2026-10-14 |
| An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 Gene therapy, Enzyme Replacement Agent - Gaucher Disease, Type 3 | Phase 2 / Phase 3 | Withdrawn | AVROBIO | 2023-10-01 | 2023-08-09 | |
| Role of Vitamin E in Gaucher Disease Patients Vitamin E - Gaucher Disease | Phase 3 | Completed | Ain Shams University | 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 | Takeda | 20 | 2023-01-03 | 2025-06-26 |
| Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 Venglustat, imiglucerase - Gaucher's Disease Type III | Phase 3 | Active not recruiting | Sanofi | 43 | 2022-04-18 | 2026-07-13 |
| 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 | Sanofi | 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 | Genzyme, a Sanofi Company | 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 | Shire | 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 | Shire | 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 | Pfizer | 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 | ISU Abxis Co., Ltd. | 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 | Pfizer | 19 | 2011-08-01 | 2023-04-19 |
| The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease Cerezyme®, ISU302 - Gaucher Disease | Phase 3 | Withdrawn | ISU Abxis Co., Ltd. | 2011-01-01 | 2016-08-23 | |
| 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 | Genzyme, a Sanofi Company | 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 | Genzyme, a Sanofi Company | 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 | Genzyme, a Sanofi Company | 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 | Pfizer | 31 | 2008-12-01 | 2018-10-04 |
| Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial Taliglucerase alfa - Gaucher Disease | Phase 3 | Completed | Pfizer | 45 | 2008-06-01 | 2018-10-04 |
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Gangliosidosis, GM1 (2) · Adrenoleukodystrophy (1) · Fucosidosis (1) · Gangliosidoses, GM2 (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Lysosomal Storage Diseases (1) · Neuronal Ceroid-Lipofuscinoses (1) · Niemann-Pick Disease, Type C (1) · Niemann-Pick Diseases (1) · Sandhoff Disease (1) · Tay-Sachs Disease (1) · Wolman Disease (1)
Brain Diseases (27) · Brain Diseases, Metabolic (27) · Brain Diseases, Metabolic, Inborn (27) · Central Nervous System Diseases (27) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (27) · Genetic Diseases, Inborn (27) · Lipid Metabolism Disorders (27) · Lipid Metabolism, Inborn Errors (27) · Lipidoses (27) · Lysosomal Storage Diseases, Nervous System (27) · Metabolic Diseases (27) · Metabolism, Inborn Errors (27) · Nervous System Diseases (27) · Nutritional and Metabolic Diseases (27) · Sphingolipidoses (27)
Glucosylceramidase (7) · imiglucerase (7) · taliglucerase alfa (6) · eliglustat (5) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Genetic Therapy (1) · Stem Cell Transplantation (1) · Velaglucerase alfa, human (1) · Vitamin E (1) · miglustat (1) · venglustat (1)
Shire (6) · Pfizer (5) · Genzyme, a Sanofi Company (4) · ISU Abxis Co., Ltd. (2) · Sanofi (2) · AVROBIO (1) · Actelion (1) · Ain Shams University (1) · Azafaros B.V. (1) · CANbridge Life Sciences Ltd. (1) · Masonic Cancer Center, University of Minnesota (1) · Spur Therapeutics (1) · Takeda (1)
United States (12) · United Kingdom (11) · Canada (10) · Israel (10) · Spain (9) · Russia (8) · Argentina (7) · Australia (5) · Japan (5) · Tunisia (5) · China (4) · France (4) · India (4) · Italy (4) · Paraguay (4)
Amel El Beshlawy, Prof. (1) · Amira A Adly, Phd (1) · Milena Veselinovic, MD, PhD (1) · Paul Orchard, MD (1) · Timothy Cox, Prof (1)
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
2008 (3), 2009 (1), 2016 (1), 2025 (1)