91 completed, 24 unknown, 23 recruiting, 9 active not recruiting. 34 Phase 2, 32 Phase 1, 27 Phase 3, 12 Not applicable, 11 Phase 4.
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 |
|---|---|---|---|---|---|---|
| A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion Gaucher Disease | Not yet recruiting | Takeda | 60 | 2027-01-31 | 2026-10-01 | |
| 4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma GD2, PSMA and CD276 CAR-T cells - Neuroblastoma | Phase 1 / Phase 2 | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 100 | 2026-12-31 | 2026-09-01 |
| 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 to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3) AZ-3102 - Gaucher Disease Type 3 | Phase 2 | Not yet recruiting | Azafaros B.V. | 20 | 2026-09-28 | 2026-07-20 |
| Nutritional Status of Gaucher Disease Patients Gaucher Disease | Not yet recruiting | Assiut University | 84 | 2026-09-26 | 2026-09-28 | |
| Parkinson's Disease in Type 1 Gaucher Disease Parkinson Disease (PD), Type 1 Gaucher Disease | Recruiting | Icahn School of Medicine at Mount Sinai | 100 | 2026-09-21 | 2026-10-27 | |
| 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 Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease VGN-R08b - Gaucher Disease Type 3 | Phase 1 / Phase 2 | Not yet recruiting | Shanghai Vitalgen BioPharma Co., Ltd. | 12 | 2026-05-14 | 2026-05-22 |
| Advancing Integrated Therapies for Gaucher Disease Gaucher Disease | Recruiting | Yale University | 30 | 2026-05-06 | 2026-08-11 | |
| 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 |
| High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3) Ambroxol - Gaucher Disease, Type 3 | Not applicable | Completed | Agyany Pharma LTD | 12 | 2025-03-01 | 2026-08-24 |
| A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease LY-M001 - Gaucher Disease | Early Phase 1 | Active not recruiting | Shanghai Jiao Tong University School of Medicine | 3 | 2024-08-01 | 2026-08-10 |
| Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3 Eliglustat Tartrate Capsules - Gaucher Disease | Phase 2 | Unknown | Peking Union Medical College Hospital | 5 | 2024-08-01 | 2024-07-26 |
| A First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male Participants YH35995, Placebo - Healthy Participants | Phase 1 | Active not recruiting | Yuhan Corporation | 94 | 2024-07-29 | 2026-07-28 |
| A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease LY-M001 - Gaucher Disease Type 1 | Phase 1 / Phase 2 | Recruiting | Lingyi Biotech Co., Ltd. | 12 | 2024-07-05 | 2026-01-26 |
| Institutional Registry of Rare Diseases Rare Diseases, Amyloidosis | Recruiting | Hospital Italiano de Buenos Aires | 380 | 2024-07-01 | 2026-01-14 | |
| FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases Alpha-Thalassemia, Beta-Thalassemia | Unknown | xCures | 1000 | 2024-06-10 | 2024-11-14 | |
| Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) FLT201 - Gaucher Disease, Type 1 | Phase 1 / Phase 2 | Active not recruiting | Spur Therapeutics | 75 | 2024-05-13 | 2025-11-04 |
| Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency Gaucher Disease | Not yet recruiting | Chung-Hsing Wang | 50 | 2024-05-01 | 2024-04-15 | |
| Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight) Investigational procedure - Gaucher Disease | Not applicable | Completed | Sanofi | 13 | 2024-04-02 | 2024-11-19 |
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Lysosomal Storage Diseases (13) · Niemann-Pick Diseases (10) · Parkinson Disease (10) · Metabolism, Inborn Errors (9) · Fabry Disease (7) · Leukodystrophy, Metachromatic (7) · Rare Diseases (7) · Wolman Disease (7) · Adrenoleukodystrophy (6) · Glycogen Storage Disease Type II (6) · Leukodystrophy, Globoid Cell (6) · Mucopolysaccharidosis I (6) · Mucopolysaccharidosis VI (6) · Sphingolipidoses (6)
Brain Diseases (175) · Brain Diseases, Metabolic (175) · Brain Diseases, Metabolic, Inborn (175) · Central Nervous System Diseases (175) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (175) · Lysosomal Storage Diseases, Nervous System (175) · Nervous System Diseases (175) · Nutritional and Metabolic Diseases (175) · Lipid Metabolism Disorders (174) · Lipid Metabolism, Inborn Errors (174) · Lipidoses (174) · Genetic Diseases, Inborn (172) · Metabolic Diseases (172) · Sphingolipidoses (169) · Metabolism, Inborn Errors (166)
Glucosylceramidase (14) · eliglustat (14) · imiglucerase (13) · taliglucerase alfa (11) · AT2101 (4) · Ambroxol (3) · miglustat (3) · Acetylcysteine (2) · Blood Specimen Collection (2) · Cyclophosphamide (2) · Enzyme Replacement Therapy (2) · Methylprednisolone (2) · Sirolimus (2) · fludarabine (2) · venglustat (2)
Shire (14) · Genzyme, a Sanofi Company (12) · Sanofi (11) · Pfizer (8) · Shaare Zedek Medical Center (7) · National Institute of Neurological Disorders and Stroke (NINDS) (6) · Takeda (6) · University of Minnesota (6) · Amicus Therapeutics (5) · ISU Abxis Co., Ltd. (5) · Lysosomal and Rare Disorders Research and Treatment Center, Inc. (5) · AVROBIO (3) · CENTOGENE GmbH Rostock (3) · National Human Genome Research Institute (NHGRI) (3) · O & O Alpan LLC (3)
United States (92) · Israel (33) · United Kingdom (24) · Spain (21) · Canada (17) · China (13) · India (13) · France (12) · Argentina (10) · Germany (10) · Russia (10) · Australia (9) · Brazil (8) · Italy (8) · Japan (8)
Ozlem Goker-Alpan, MD (6) · Ari Zimran, MD (3) · Renuka Limgala, PhD (3) · Ari Zimran (2) · Ari Zimran, Prof. (2) · Ellen Sidransky, M.D. (2) · John Barranger (2) · Milena Veselinovic, MD (2) · Paul Szabolcs, MD (2) · Paul Tuite, MD (2) · A. Jon Stoessl, CM, MD, FRCPC, FCAHS (1)
PMID 27839979, PMID 23860343, PMID 26053270, PMID 36739645, PMID 41527340, PMID 14578207, PMID 15324318, PMID 1603628, PMID 16546416, PMID 17230068, PMID 18729283, PMID 19277123, PMID 19948615, PMID 20299511, PMID 20439622, PMID 20713962, PMID 21586746, PMID 21637107, PMID 21799847, PMID 21900191, PMID 23199589, PMID 23270487, PMID 23339116, PMID 23510062, PMID 23520473, PMID 24263462, PMID 24488939, PMID 24816856, PMID 24835462, PMID 24924293, PMID 24950666, PMID 25409372, PMID 25453586, PMID 25688781, PMID 25801797, PMID 25819691, PMID 25829804, PMID 25994334, PMID 26043810, PMID 26051481, PMID 26096740, PMID 26096743, PMID 26781431, PMID 27102949, PMID 27174694, PMID 27241455, PMID 27499018, PMID 27839981, PMID 27866808, PMID 28167660
2008 (3), 2019 (2), U54NS065768 (2), 2005 (1), 2009 (1), 2014 (1), 2015 (1), 2016 (1), 2023 (1), 2025 (1), K12CA120780 (1), R01HD073292 (1)