37 completed, 7 terminated, 5 withdrawn, 4 recruiting. 61 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 61 | Completed 37, Terminated 7, Withdrawn 5, Recruiting 4, Unknown 4, Active not recruiting 2, Enrolling by invitation 1, Not yet recruiting 1 |
Follow Lysosomal Storage Diseases: new trials by email Literature Review of Lysosomal Storage Diseases trials Research Report on Lysosomal Storage Diseases
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 |
|---|---|---|---|---|---|---|
| China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD Avalglucosidase alfa - Glycogen Storage Disease Type II, Pompe's Disease | Phase 4 | Recruiting | Genzyme, a Sanofi Company | 13 | 2025-05-07 | 2026-03-23 |
| Treatment Frequency Reduction in Pompe Disease Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks - Pompe Disease (Late-onset), GAA Deficiency | Phase 4 | Not yet recruiting | Erasmus Medical Center | 10 | 2024-10-01 | 2024-08-28 |
| 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 |
| Comparing Efficacy of 8-Week and 12-Week Faricimab Initial Follow-Up Treatment Intervals Faricimab Injection - Wet Age-related Macular Degeneration | Phase 4 | Recruiting | Faculty Hospital Kralovske Vinohrady | 100 | 2023-03-31 | 2025-03-13 |
| A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II) ELAPRASE, Rituximab, Methotrexate - Mucopolysaccharidosis (MPS), Hunter Syndrome | Phase 4 | Withdrawn | Takeda | 2023-02-28 | 2025-12-09 | |
| Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE AX 250 - MPS III B | Phase 4 | Enrolling by invitation | Allievex Corporation | 15 | 2022-12-02 | 2023-08-09 |
| 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 |
| Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years Avalglucosidase Alfa - Glycogen Storage Disease Type II Late Onset, Pompe Disease (Late-onset) | Phase 4 | Active not recruiting | Iris Plug | 6 | 2022-10-13 | 2026-06-17 |
| Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) Avalglucosidase alfa (GZ402666) - Glycogen Storage Disease Type II | Phase 4 | Active not recruiting | Genzyme, a Sanofi Company | 17 | 2022-07-11 | 2026-09-09 |
| A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India Elaprase - Hunter Syndrome | Phase 4 | Completed | Takeda | 5 | 2022-04-21 | 2025-01-23 |
| China Post-marketing Surveillance (PMS) Study of Aldurazyme® Laronidase - Mucopolysaccharidosis I | Phase 4 | Completed | Genzyme, a Sanofi Company | 12 | 2021-10-28 | 2025-09-17 |
| 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 |
| Awareness in Old Aged Patients During Laryngoscopy and Intubation Using Isolated Forearm Technique Sevoflurane, TIVA - Awareness, Anesthesia, Recall Phenomenon | Phase 4 | Completed | Ain Shams University | 50 | 2021-09-05 | 2022-01-13 |
| 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 in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen ALGLUCOSIDASE ALFA - Pompe's Disease | Phase 4 | Completed | Genzyme, a Sanofi Company | 41 | 2021-03-10 | 2025-02-10 |
| 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 |
| Effects of Bisphosphonates on OI-Related Hearing Loss Risedronate Oral Tablet - Osteogenesis Imperfecta | Phase 4 | Recruiting | Hospital for Special Surgery, New York | 100 | 2019-11-02 | 2026-04-28 |
Showing 20 of 61. Sign in to see all of them.
Glycogen Storage Disease Type II (19) · Fabry Disease (13) · Gaucher Disease (11) · Mucopolysaccharidosis IV (5) · Mucopolysaccharidosis I (4) · Mucopolysaccharidosis II (4) · Abdominal Pain (1) · Brittle Bone Disorder (1) · Cardiomyopathies (1) · Constipation (1) · Cystinosis (1) · Diarrhea (1) · Gangliosidoses (1) · Gangliosidoses, GM2 (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (61) · Genetic Diseases, Inborn (61) · Metabolic Diseases (61) · Metabolism, Inborn Errors (61) · Nutritional and Metabolic Diseases (61) · Nervous System Diseases (50) · Brain Diseases (45) · Brain Diseases, Metabolic (45) · Brain Diseases, Metabolic, Inborn (45) · Central Nervous System Diseases (45) · Lysosomal Storage Diseases, Nervous System (45) · Carbohydrate Metabolism, Inborn Errors (34) · Lipid Metabolism Disorders (26) · Lipid Metabolism, Inborn Errors (26)
GAA protein, human (12) · agalsidase beta (10) · agalsidase alfa (5) · Iduronidase (4) · idursulfase (4) · Glucosylceramidase (3) · Methotrexate (3) · Rituximab (3) · imiglucerase (3) · Immunoglobulins, Intravenous (2) · miglustat (2) · taliglucerase alfa (2) · Acetaminophen (1) · Acetylcysteine (1) · Albuterol (1)
Genzyme, a Sanofi Company (26) · Shire (4) · National Taiwan University Hospital (3) · Sanofi (3) · Takeda (3) · Pfizer (2) · Actelion (1) · Ain Shams University (1) · Allievex Corporation (1) · Ari Zimran (1) · Assistance Publique - Hôpitaux de Paris (1) · Baylor Research Institute (1) · BioMarin Pharmaceutical (1) · Clínica de Hidroterapia e Recuperacao Funcional (1) · Copenhagen University Hospital, Rigshospitalet (1)
United States (31) · United Kingdom (8) · China (7) · Canada (6) · Taiwan (5) · France (4) · Germany (4) · India (4) · Israel (4) · Poland (4) · Brazil (3) · Czechia (3) · Netherlands (3) · Australia (2) · Egypt (2)
Yin-Hsiu Chien, MD, PhD (2) · Ari Zimran (1) · Ari Zimran, Prof. (1) · Cathleen Raggio, MD (1) · Celeste Decker, MD (1) · Edward Kaye, M.D. (1) · Jeanine R. Jarnes, PharmD (1) · Maria Helena Vaisbich, Doctor (1) · Martin Pencak, MD (1)
PMID 17179052, PMID 21596994, PMID 22393902, PMID 24108527, PMID 25453586, PMID 25864295, PMID 25902842, PMID 27473031, PMID 28476546, PMID 29565424, PMID 29989200, PMID 31086307, PMID 33775523, PMID 34420548, PMID 35833019, PMID 36214004, PMID 37705424, PMID 38715621, PMID 39628618, PMID 40760696, PMID 41163043
2015 (2), 2019 (2), 1RC1DK086182 (1), 2014 (1), R01DK067071 (1), R01DK079866 (1), U54NS065768 (1), UL1RR024150 (1)