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 | EUCTR2005-003512-30-FR (EUCTR) | 05/04/2007 | 26/03/2007 | A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A | A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A | Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome) MedDRA version: 6.0;Level: PT;Classification code 10056892 | Trade Name: Naglazyme Product Name: Naglazyme INN or Proposed INN: galsulfase Other descriptive name: recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 4 | Phase 4 | Portugal;France | ||
| 2 | NCT00104234 (ClinicalTrials.gov) | February 2004 | 24/2/2005 | Study of rhASB in Patients With Mucopolysaccharidosis VI | A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI | Mucopolysaccharidosis VI | Drug: N-acetylgalactosamine 4-sulfatase;Drug: Placebo/rhASB | BioMarin Pharmaceutical | NULL | Completed | 7 Years | N/A | All | 39 | Phase 3 | United States |
| 3 | NCT00067470 (ClinicalTrials.gov) | September 2003 | 20/8/2003 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI | Mucopolysaccharidosis VI | Drug: Placebo;Drug: N-acetylgalactosamine 4-sulfatase | BioMarin Pharmaceutical | NULL | Completed | 7 Years | N/A | All | 39 | Phase 3 | United States | |
| 4 | NCT00048711 (ClinicalTrials.gov) | March 2002 | 6/11/2002 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | Mucopolysaccharidosis VI | Drug: N-acetylgalactosamine 4-sulfatase | BioMarin Pharmaceutical | NULL | Completed | 5 Years | N/A | Both | Phase 2 | United States | |
| 5 | NCT00048620 (ClinicalTrials.gov) | September 2000 | 4/11/2002 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | Mucopolysaccharidosis VI | Drug: N-acetylgalactosamine 4-sulfatase | BioMarin Pharmaceutical | NULL | Completed | N/A | N/A | Both | Phase 1 | United States | |
| 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 |
| 6 | EUCTR2004-000642-21-IE (EUCTR) | 18/06/2004 | A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI - Aryplase Phase 3 Open-Label Extension | A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI - Aryplase Phase 3 Open-Label Extension | Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome) MedDRA version: 6.0;Level: PT;Classification code 10056892 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 38 | Phase 3 | Ireland |