84 completed. 84 Phase 3, 15 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 84 | Completed 84 |
| Phase 2 | 15 | Completed 15 |
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 |
|---|---|---|---|---|---|---|
| A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day - Achondroplasia | Phase 3 | Completed | QED Therapeutics, a BridgeBio company | 114 | 2023-11-10 | 2026-03-17 |
| Role of Vitamin E in Gaucher Disease Patients Vitamin E - Gaucher Disease | Phase 3 | Completed | Ain Shams University | 40 | 2023-03-03 | 2024-01-18 |
| A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease Velaglucerase Alfa - Gaucher Disease | Phase 3 | Completed | Takeda | 20 | 2023-01-03 | 2025-06-26 |
| Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease Recombinant human alpha galactosidase A (agalsidase beta), Recombinant human alpha-galactosidase A (agalsidase beta) - Fabry Disease | Phase 3 | Completed | Bio Sidus SA | 20 | 2022-12-13 | 2026-02-18 |
| Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients Laronidase, Antihistamine, Antipyretic - Mucopolysaccharidosis Type 1 | Phase 3 | Completed | Cinnagen | 12 | 2022-09-10 | 2024-08-06 |
| A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease REPLAGAL - Fabry Disease | Phase 3 | Completed | Takeda | 20 | 2022-05-01 | 2024-10-08 |
| Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) Mercaptamine - Cystinosis | Phase 3 | Completed | RECORDATI GROUP | 5 | 2020-09-01 | 2024-02-05 |
| Efficacy and Safety of mAnnitol in Bowel Preparation During Elective Colonoscopy and Comparison With Moviprep® Phase II: NTC015 low dose, Phase II: NTC015 medium dose, Phase II: NTC015 high dose - Elective Colonoscopy | Phase 2 / Phase 3 | Completed | NTC srl | 886 | 2020-06-18 | 2025-12-08 |
| A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) AT2221, ATB200 - Pompe Disease (Late-onset) | Phase 3 | Completed | Amicus Therapeutics | 119 | 2019-12-18 | 2026-03-05 |
| Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease migalastat HCl 150 mg - Fabry Disease | Phase 3 | Completed | Amicus Therapeutics | 16 | 2019-10-11 | 2026-02-05 |
| A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease Lucerastat - Fabry Disease | Phase 3 | Completed | Idorsia Pharmaceuticals Ltd. | 107 | 2018-12-18 | 2026-11-02 |
| A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa - Pompe Disease (Late-onset) | Phase 3 | Completed | Amicus Therapeutics | 125 | 2018-12-04 | 2025-09-11 |
| Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients pegunigalsidase alfa - Fabry Disease | Phase 3 | Completed | Chiesi Farmaceutici S.p.A. | 29 | 2018-11-13 | 2026-05-22 |
| Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) Migalastat HCl 150 mg - Fabry Disease | Phase 3 | Completed | Amicus Therapeutics | 22 | 2018-09-27 | 2021-11-30 |
| Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease pegunigalsidase alfa - Fabry Disease | Phase 3 | Completed | Chiesi Farmaceutici S.p.A. | 97 | 2018-09-16 | 2026-03-24 |
| A Study of JR-141 in Patients With Mucopolysaccharidosis II JR-141 - Mucopolysaccharidosis II | Phase 2 / Phase 3 | Completed | JCR Pharmaceuticals Co., Ltd. | 28 | 2018-08-01 | 2022-11-14 |
| Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease Lucerastat, Placebo - Fabry Disease | Phase 3 | Completed | Idorsia Pharmaceuticals Ltd. | 118 | 2018-06-21 | 2024-08-09 |
| An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta) Hunterase - MPS II, Hunter Syndrome (MPS II) | Phase 3 | Completed | GC Biopharma Corp | 30 | 2018-05-23 | 2026-01-15 |
| Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 Eliglustat GZ385660, Imiglucerase GZ437843 - Gaucher's Disease Type I, Gaucher's Disease Type III | Phase 3 | Completed | Sanofi | 57 | 2018-04-11 | 2026-06-16 |
| Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks Pegunigalsidase alfa - Fabry Disease | Phase 3 | Completed | Protalix | 30 | 2017-07-10 | 2023-09-13 |
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Gaucher Disease (21) · Fabry Disease (19) · Mucopolysaccharidosis II (8) · Glycogen Storage Disease Type II (6) · Mucopolysaccharidosis IV (6) · Rare Diseases (6) · Cystinosis (5) · Sudden Infant Death (5) · Metabolic Diseases (4) · Mucopolysaccharidosis I (4) · alpha-Mannosidosis (4) · Niemann-Pick Disease, Type C (3) · Proteinuria (3) · Wolman Disease (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (84) · Metabolism, Inborn Errors (84) · Nutritional and Metabolic Diseases (84) · Genetic Diseases, Inborn (83) · Metabolic Diseases (80) · Nervous System Diseases (60) · Brain Diseases (52) · Brain Diseases, Metabolic (52) · Brain Diseases, Metabolic, Inborn (52) · Central Nervous System Diseases (52) · Lysosomal Storage Diseases, Nervous System (52) · Lipid Metabolism Disorders (47) · Lipid Metabolism, Inborn Errors (47) · Lipidoses (47)
Glucosylceramidase (7) · migalastat (7) · GAA protein, human (5) · agalsidase alfa (5) · agalsidase beta (5) · eliglustat (5) · larazotide acetate (5) · miglustat (5) · taliglucerase alfa (5) · Cysteamine (4) · imiglucerase (4) · Iduronidase (3) · idursulfase (3) · Chondroitinsulfatases (2) · GALNS protein, human (2)
Genzyme, a Sanofi Company (14) · Shire (13) · Amicus Therapeutics (7) · BioMarin Pharmaceutical (5) · Pfizer (5) · Amgen (4) · Chiesi Farmaceutici S.p.A. (4) · Protalix (3) · Alexion Pharmaceuticals, Inc. (2) · Green Cross Corporation (2) · Idorsia Pharmaceuticals Ltd. (2) · Takeda (2) · Ultragenyx Pharmaceutical Inc (2) · Zymenex A/S (2) · Actelion (1)
United States (53) · United Kingdom (44) · France (34) · Canada (26) · Italy (25) · Spain (25) · Australia (22) · Germany (19) · Netherlands (19) · Argentina (16) · Brazil (14) · Japan (14) · Denmark (13) · Poland (13) · Belgium (11)
Allan M Lund, MD (2) · Debra Lounsbury (2) · Ali Rabani, Professor (1) · Amel El Beshlawy, Prof. (1) · Amira A Adly, Phd (1) · Chester Whitley, MD (1)
PMID 33874971, PMID 27839979, PMID 39847314, PMID 19775921, PMID 26053270, PMID 27657681, PMID 31889231, PMID 32063397, PMID 32994552, PMID 35961250, PMID 37415957, PMID 37915038, PMID 39774103, PMID 41631150, PMID 18729283, PMID 19117887, PMID 20393176, PMID 20730727, PMID 21150784, PMID 21543987, PMID 21586746, PMID 21900191, PMID 22476655, PMID 22929184, PMID 23270487, PMID 23339116, PMID 23531228, PMID 23837440, PMID 24948347, PMID 24950666, PMID 24993530, PMID 25487082, PMID 25688781, PMID 25795794, PMID 25801797, PMID 25819691, PMID 25955246, PMID 26043810, PMID 26185417, PMID 27102949, PMID 27174694, PMID 27241455, PMID 27499018, PMID 27509102, PMID 27834756, PMID 27839981, PMID 27955919, PMID 28167660, PMID 28535791, PMID 29248359
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