7 completed, 4 unknown, 1 recruiting. 12 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 12 | Completed 7, Unknown 4, Recruiting 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I LY-M001 Injection - Gaucher Disease Type I | Not applicable | Recruiting | He Huang | 3 | 2023-11-15 | 2025-02-14 |
| Getting Global Rare Disease Insights Through Technology Study Zamplo Digital Health Platform - Metabolic Disease, Fabry Disease | Not applicable | Unknown | M.A.G.I.C. Clinic LTD | 150 | 2020-08-07 | 2021-04-08 |
| Therapeutic Exercise to Treat Neuropathic Pain Therapeutic Exercise - Gaucher Disease, Fabry Disease | Not applicable | Completed | Universidad de Zaragoza | 5 | 2019-05-02 | 2020-12-16 |
| Eliglustat on Gaucher Disease Type IIIB Eliglustat - Gaucher Disease, Type III | Not applicable | Completed | National Taiwan University Hospital | 4 | 2018-04-23 | 2022-09-15 |
| Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs quantitative chemical shift imaging (QCSI), Elelyso - Gaucher Disease, Type 1 | Not applicable | Completed | Shaare Zedek Medical Center | 30 | 2017-01-01 | 2022-10-28 |
| Miglustat on Gaucher Disease Type IIIB Miglustat, ERT - Gaucher Disease | Not applicable | Unknown | National Taiwan University Hospital | 19 | 2015-07-01 | 2019-03-29 |
| Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease BeamMed Speed-of-Sound bone strength test at forearm - Gaucher Disease Type 1 | Not applicable | Completed | Shaare Zedek Medical Center | 100 | 2014-02-01 | 2014-11-19 |
| Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan Fibroscan - Fibrosis | Not applicable | Unknown | Rambam Health Care Campus | 40 | 2013-08-01 | 2013-07-12 |
| Identification of Undiagnosed Gaucher Disease Enzyme analysis - Gaucher Disease | Not applicable | Unknown | Brigham and Women's Hospital | 100 | 2012-08-01 | 2012-10-30 |
| Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease Bone marrow and blood sampling - Gaucher Disease | Not applicable | Completed | Assistance Publique - Hôpitaux de Paris | 30 | 2009-12-01 | 2014-05-23 |
Fabry Disease (2) · Lysosomal Storage Diseases (2) · Metabolic Diseases (2) · Ataxia (1) · Fibrosis (1) · Glycogen Storage Disease Type II (1) · Mitochondrial Diseases (1) · Motor Activity (1) · Myoclonus (1) · Neurologic Manifestations (1)
Brain Diseases (12) · Brain Diseases, Metabolic (12) · Brain Diseases, Metabolic, Inborn (12) · Central Nervous System Diseases (12) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Genetic Diseases, Inborn (12) · Lipid Metabolism Disorders (12) · Lipid Metabolism, Inborn Errors (12) · Lipidoses (12) · Lysosomal Storage Diseases, Nervous System (12) · Metabolism, Inborn Errors (12) · Nervous System Diseases (12) · Nutritional and Metabolic Diseases (12) · Sphingolipidoses (12) · Lysosomal Storage Diseases (10)
Ambroxol (1) · Blood Specimen Collection (1) · Exercise Therapy (1) · Restriction Mapping (1) · eliglustat (1) · miglustat (1) · taliglucerase alfa (1)
National Taiwan University Hospital (2) · Shaare Zedek Medical Center (2) · Agyany Pharma LTD (1) · Assistance Publique - Hôpitaux de Paris (1) · Brigham and Women's Hospital (1) · He Huang (1) · M.A.G.I.C. Clinic LTD (1) · Rambam Health Care Campus (1) · Sanofi (1) · Universidad de Zaragoza (1)
Taiwan (2) · United States (2) · Canada (1) · China (1) · France (1) · Israel (1) · Pakistan (1) · Spain (1)
Ari Zimran, MD (1) · Ari Zimran, Prof. (1) · Bertrabd Arnulf, MD (1) · Hanna Rosenbaum (1) · Huma Arshad Cheema, Prof. (1) · Michael F Murray, MD (1) · Ni-Chung Lee, M.D., Ph.D (1) · Yin-Hsiu Chien, M.D., Ph.D. (1)
PMID 14578207, PMID 1603628, PMID 17230068, PMID 23199589, PMID 23520473, PMID 24488939, PMID 24924293, PMID 25409372, PMID 26781431, PMID 28621801, PMID 29719368, PMID 30066229, PMID 30622983, PMID 32146279, PMID 33067633, PMID 35857660