19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | Not Recruiting | 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 | |
| 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 | Product Name: recombinant human N-acetylgalactosamine 4-sulfatase Product Code: N/A | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 38 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): | Ireland |