18 completed. 18 Phase 1, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 18 | Completed 18 |
| Phase 2 | 2 | Completed 2 |
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 Gene Therapy Study in Patients With Gaucher Disease Type 1 FLT201 - Gaucher Disease, Type 1 | Phase 1 | Completed | Spur Therapeutics | 10 | 2022-04-15 | 2026-06-02 |
| A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers Abcertin, EU-sourced Cerezyme - Gaucher Disease | Phase 1 | Completed | ISU Abxis Co., Ltd. | 42 | 2020-01-29 | 2021-04-05 |
| A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers Eliglustat - Gaucher's Disease | Phase 1 | Completed | Sanofi | 18 | 2018-01-01 | 2024-01-10 |
| A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate eliglustat - Gaucher Disease | Phase 1 | Completed | Genzyme, a Sanofi Company | 24 | 2015-09-01 | 2017-02-10 |
| A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate eliglustat - Gaucher Disease | Phase 1 | Completed | Sanofi | 32 | 2015-09-01 | 2017-03-08 |
| Taste Evaluation of Different Liquid Formulations With Eliglustat eliglustat - Gaucher Disease | Phase 1 | Completed | Genzyme, a Sanofi Company | 8 | 2015-04-01 | 2015-05-27 |
| A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth Eliglustat - Gaucher's Disease | Phase 1 | Completed | Sanofi | 6 | 2014-08-25 | 2024-01-05 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients PRX-112 - Gaucher Disease | Phase 1 | Completed | Protalix | 16 | 2013-03-01 | 2014-04-07 |
| Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease velaglucerase alfa - Gaucher Disease, Type 3 | Phase 1 / Phase 2 | Completed | Shire | 7 | 2012-09-14 | 2021-06-11 |
| Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease N-acetylcysteine - Parkinson's Disease, Gaucher's Disease | Phase 1 | Completed | University of Minnesota | 9 | 2011-07-01 | 2019-11-01 |
| A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers ISU302, Placebo - Gaucher Disease | Phase 1 | Completed | ISU Abxis Co., Ltd. | 24 | 2010-10-01 | 2013-06-19 |
| A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants Genz-112638, [14C]-Genz-112638 - Gaucher's Disease Type I | Phase 1 | Completed | Sanofi | 10 | 2009-06-03 | 2023-11-22 |
| Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD) Human Glucocerebrosidase (prGCD) - Gaucher Disease | Phase 1 | Completed | Protalix | 6 | 2005-11-01 | 2006-12-05 |
| Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) GA-GCB - Gaucher Disease | Phase 1 / Phase 2 | Completed | Shire | 10 | 2004-09-13 | 2021-06-22 |
| Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease human glucocerebrosidase gene into autologous peripheral blood stem cells - Gaucher's Disease | Phase 1 | Completed | National Center for Research Resources (NCRR) | 15 | 1999-11-01 | 2005-06-24 |
| PEG-Glucocerebrosidase for the Treatment of Gaucher Disease Lysodase - Gaucher's Disease | Phase 1 | Completed | National Institute of Mental Health (NIMH) | 18 | 1993-10-01 | 2008-03-04 |
| Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells human glucocerebrosidase cDNA - Gaucher's Disease | Phase 1 | Completed | National Institute of Neurological Disorders and Stroke (NINDS) | 120 | 1988-01-01 | 2008-03-04 |
Metabolism, Inborn Errors (3) · Adrenoleukodystrophy (1) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1) · Fucosidosis (1) · Genetic Diseases, Inborn (1) · Leukemia, Myeloid, Acute (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis VI (1) · Myelodysplastic Syndromes (1) · Neuronal Ceroid-Lipofuscinoses (1) · Niemann-Pick Diseases (1)
Brain Diseases (18) · Brain Diseases, Metabolic (18) · Brain Diseases, Metabolic, Inborn (18) · Central Nervous System Diseases (18) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Lipid Metabolism Disorders (18) · Lipid Metabolism, Inborn Errors (18) · Lipidoses (18) · Lysosomal Storage Diseases (18) · Lysosomal Storage Diseases, Nervous System (18) · Metabolic Diseases (18) · Nervous System Diseases (18) · Nutritional and Metabolic Diseases (18) · Genetic Diseases, Inborn (17) · Sphingolipidoses (17)
eliglustat (6) · Glucosylceramidase (2) · Abcertin (1) · Acetylcysteine (1) · Enzyme Replacement Therapy (1) · imiglucerase (1) · taliglucerase alfa (1)
Sanofi (4) · Genzyme, a Sanofi Company (2) · ISU Abxis Co., Ltd. (2) · Protalix (2) · Shire (2) · National Center for Research Resources (NCRR) (1) · National Institute of Mental Health (NIMH) (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · New York Medical College (1) · Spur Therapeutics (1) · University of Minnesota (1)
United States (11) · Israel (3) · Australia (1) · Brazil (1) · Egypt (1) · Germany (1) · India (1) · Romania (1) · Serbia (1) · Spain (1) · Tunisia (1) · United Kingdom (1)
Charlotte Lemech, MD (1) · Einat Almon, PhD (1) · Eithan Galun, MD (1) · John Barranger (1) · Mitchell S Cairo, MD (1) · Paul Tuite, MD (1)
PMID 19277123, PMID 20299511, PMID 23860343
U54NS065768 (1)