19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | NCT00813865 (ClinicalTrials.gov) | May 11, 2009 | 22/12/2008 | A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients | An Open-Label, Multicenter, Long-Term Extension Study To Assess The Safety, Efficacy, And Pharmacodynamics Of AT2101 In Adult Patients With Type 1 Gaucher Disease | Gaucher Disease;Type 1 Gaucher Disease;Gaucher Disease, Type 1 | Drug: afegostat tartrate | Amicus Therapeutics | NULL | Completed | 18 Years | N/A | All | 8 | Phase 2 | United States;United Kingdom;Paraguay |
2 | EUCTR2008-007158-36-GB (EUCTR) | 25/02/2009 | 29/04/2009 | An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease. | An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease. | Type I Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: afegostat tartrate Product Code: AT2101 INN or Proposed INN: afegostat tartrate | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 16 | United Kingdom | |||
3 | NCT00446550 (ClinicalTrials.gov) | June 11, 2008 | 9/3/2007 | A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease | A Randomized, Open-label Study To Assess the Safety and Tolerability of AT2101 in Treatment-naive Adult Patients With Type 1 Gaucher Disease | Gaucher Disease;Type 1 Gaucher Disease;Gaucher Disease, Type 1 | Drug: afegostat tartrate | Amicus Therapeutics | NULL | Completed | 18 Years | 74 Years | All | 19 | Phase 2 | United States;Israel;South Africa;United Kingdom;Germany |
4 | NCT00433147 (ClinicalTrials.gov) | March 23, 2007 | 7/2/2007 | A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy | A Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy | Gaucher Disease, Type 1;Type 1 Gaucher Disease;Gaucher Disease | Drug: Afegostat tartrate | Amicus Therapeutics | NULL | Completed | 18 Years | 74 Years | All | 30 | Phase 2 | United States |