18 completed, 4 withdrawn, 2 terminated, 2 unknown. 27 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 27 | Completed 18, Withdrawn 4, Terminated 2, Unknown 2, Recruiting 1 |
Follow Lipidoses: new trials by email Literature Review of Lipidoses trials Research Report on Lipidoses
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 Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine - Fabry's Disease | Phase 4 | Completed | Sanofi | 8 | 2023-11-10 | 2025-10-21 |
| A Study of Replagal in Children and Adults With Fabry Disease in India Replagal - Fabry Disease | Phase 4 | Recruiting | Shire | 5 | 2022-11-01 | 2026-09-03 |
| China Post-marketing Surveillance (PMS) Study of Fabrazyme® Agalsidase beta - Fabry Disease | Phase 4 | Completed | Genzyme, a Sanofi Company | 22 | 2021-10-13 | 2025-09-11 |
| A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy Digital Engagement Application (GD App), No Intervention - Gaucher Disease | Phase 4 | Completed | Takeda | 4 | 2021-04-22 | 2023-12-21 |
| Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ Cerezyme® / Imiglucerase - Gaucher's Disease | Phase 4 | Completed | Sanofi | 12 | 2021-03-02 | 2025-09-15 |
| A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease Elelyso - Gaucher Disease, Type 3 | Phase 4 | Completed | Ari Zimran | 14 | 2020-11-20 | 2024-12-17 |
| To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease agalsidase beta (GZ419828), agalsidase alfa - Fabry Disease | Phase 4 | Withdrawn | Sanofi | 2020-09-01 | 2023-04-07 | |
| Safety and Efficacy of Miglustat in Chinese NPC Patients Miglustat - Niemann-Pick Disease, Type C | Phase 4 | Completed | Actelion | 17 | 2020-04-02 | 2025-03-30 |
| Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease Elelyso 60 units/kg - Type 1 Gaucher Disease | Phase 4 | Withdrawn | Pfizer | 2019-07-31 | 2019-09-24 | |
| Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease. Ledipasvir/Sofosbuvir - Gaucher Disease, HCV | Phase 4 | Unknown | Mansoura University Children Hospital | 10 | 2018-04-03 | 2018-10-30 |
| Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease Velaglucerase alfa, Vitamin D - Gaucher Disease | Phase 4 | Completed | Shire | 21 | 2016-06-29 | 2022-02-01 |
| Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) VPRIV - Gaucher Disease, Type 1 | Phase 4 | Completed | Shaare Zedek Medical Center | 15 | 2016-01-10 | 2022-10-28 |
| Synergistic Enteral Regimen for Treatment of the Gangliosidoses miglustat, Ketogenic Diet - GM1 Gangliosidoses, GM2 Gangliosidoses | Phase 4 | Terminated | University of Minnesota | 16 | 2015-12-22 | 2021-04-14 |
| The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease Velaglucerase alfa - Gaucher Disease Type 1, Gaucher Disease Type 3 | Phase 4 | Withdrawn | Baylor Research Institute | 2015-07-01 | 2018-03-02 | |
| A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta Agalsidase beta - Fabry Disease | Phase 4 | Completed | Genzyme, a Sanofi Company | 15 | 2012-04-01 | 2014-07-10 |
| A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease Taliglucerase alfa - Gaucher Disease | Phase 4 | Completed | Pfizer | 11 | 2010-08-01 | 2018-10-05 |
| Study of the Effects of Fabrazyme Treatment on Lactation and Infants agalsidase beta - Fabry Disease, Alpha Galactosidase A Deficiency | Phase 4 | Terminated | Genzyme, a Sanofi Company | 7 | 2006-05-28 | 2024-04-10 |
| A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency. Fabrazyme (agalsidase beta) - Fabry Disease | Phase 4 | Withdrawn | Genzyme, a Sanofi Company | 2006-04-01 | 2014-02-05 | |
| A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease Agalsidase beta - Fabry Disease | Phase 4 | Completed | Genzyme, a Sanofi Company | 6 | 2005-07-01 | 2015-05-12 |
| Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease recombinant alpha-galactosidase A - Fabry Disease | Phase 4 | Unknown | Assistance Publique - Hôpitaux de Paris | 34 | 2005-06-01 | 2007-06-18 |
Showing 20 of 27. Sign in to see all of them.
Fabry Disease (13) · Gaucher Disease (11) · Brain Diseases (1) · Cardiomyopathies (1) · Ceroid lipofuscinosis, neuronal 1, infantile (1) · Gangliosidoses (1) · Gangliosidoses, GM2 (1) · Gangliosidosis, GM1 (1) · Lysosomal Storage Diseases (1) · Neuronal Ceroid-Lipofuscinoses (1) · Niemann-Pick Disease, Type C (1) · Sandhoff Disease (1) · Stroke (1) · Tay-Sachs Disease (1) · Tay-Sachs Disease, Juvenile (1)
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) · Metabolic Diseases (27) · Metabolism, Inborn Errors (27) · Nervous System Diseases (27) · Nutritional and Metabolic Diseases (27) · Brain Diseases (26) · Brain Diseases, Metabolic (26) · Brain Diseases, Metabolic, Inborn (26) · Lysosomal Storage Diseases, Nervous System (26) · Sphingolipidoses (26)
agalsidase beta (10) · agalsidase alfa (5) · Glucosylceramidase (3) · imiglucerase (3) · miglustat (2) · taliglucerase alfa (2) · Acetaminophen (1) · Cetirizine (1) · Cysteamine (1) · Dexamethasone (1) · Diet, Ketogenic (1) · Diphenhydramine (1) · Enzyme Replacement Therapy (1) · Injections (1) · Loratadine (1)
Genzyme, a Sanofi Company (9) · Sanofi (3) · Pfizer (2) · Shire (2) · Actelion (1) · Ari Zimran (1) · Assistance Publique - Hôpitaux de Paris (1) · Baylor Research Institute (1) · Copenhagen University Hospital, Rigshospitalet (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Mansoura University Children Hospital (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · Shaare Zedek Medical Center (1) · Takeda (1) · University of Minnesota (1)
United States (15) · United Kingdom (5) · Canada (3) · China (3) · Israel (3) · Poland (3) · Czechia (2) · Hungary (2) · India (2) · Spain (2) · Austria (1) · Brazil (1) · Denmark (1) · Egypt (1) · France (1)
Anil B Mukherjee, M.D. (1) · Ari Zimran (1) · Ari Zimran, Prof. (1) · Edward Kaye, M.D. (1) · Jeanine R. Jarnes, PharmD (1) · Raphael Schiffmann, M.D.,M.H.Sc. (1) · Ulla Feldt-Rasmussen, MD, DMSc (1) · Yue Wu (1)
PMID 17179052, PMID 24997880, PMID 25453586, PMID 28476546, PMID 29989200, PMID 39628618, PMID 40760696
2015 (1), 2019 (1), U54NS065768 (1)