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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2008-005825-12-IT (EUCTR) | 28/07/2009 | 16/09/2009 | : A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy - PB-06-002 | : A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy - PB-06-002 | Gaucher disease MedDRA version: 9.1;Level: SOC;Classification code 10029205 | Product Name: Plant Cell Recombinant Glucocerebrosidase Product Code: prGCD | PROTALIX BIOTHERAPEUTICS | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 3 | Germany;United Kingdom;Spain;Italy | ||
| 2 | EUCTR2008-005826-35-IT (EUCTR) | 19/06/2009 | 30/06/2009 | A multicenter, double-blind, extension trial of two parallel dose groups of Plant cell expressed recombinant human glucocerebrosidase (prGCD) in patients with Gaucher disiease. - PB-06-003 | A multicenter, double-blind, extension trial of two parallel dose groups of Plant cell expressed recombinant human glucocerebrosidase (prGCD) in patients with Gaucher disiease. - PB-06-003 | Gaucher Disease | Product Name: Plant Cell Recombinant Glucocerebrosidase Product Code: prGCD Product Name: Plant Cell Recombinant Glucocerebrosidase Product Code: prGCD | PROTALIX BIOTHERAPEUTICS | NULL | Not Recruiting | Female: yes Male: yes | 30 | United Kingdom;Spain;Italy | |||
| 3 | EUCTR2007-000498-42-IT (EUCTR) | 13/11/2008 | 19/03/2009 | A phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two parallel Dose Groups of Plant CElle Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease - ND | A phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two parallel Dose Groups of Plant CElle Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease - ND | Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Plant Cell Recombinant Glucocerebrosidase Product Code: prGCD INN or Proposed INN: GLUCOCEREBROSIDASE Product Name: Plant Cell Recombinant Glucocerebrosidase Product Code: prGCD INN or Proposed INN: GLUCOCEREBROSIDASE | PROTALIX BIOTHERAPEUTICS | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United Kingdom;Spain;Italy |