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 | EUCTR2010-020199-45-DE (EUCTR) | 14/06/2012 | 12/09/2011 | A phase 3 extension study to evaluate the long term efficacy and safety of BMN 110 in patients with Mucopolysaccharidosis IV A (Morquio A syndrome) | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) - NA | Mucopolysaccharidosis type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: recombinant human Nacetylgalactosamine- Product Code: BMN 110 INN or Proposed INN: Not Available Other descriptive name: recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Turkey;Colombia;Italy;Switzerland;United Kingdom;France;Canada;Argentina;Brazil;Poland;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of | ||
| 2 | EUCTR2010-020199-45-PT (EUCTR) | 02/03/2012 | 29/12/2011 | A phase 3 extension study to evaluate the long term efficacy and safety of BMN 110 in patients with Mucopolysaccharidosis IV A (Morquio A syndrome) | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) - NA | Mucopolysaccharidosis type IVA MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: recombinant human Nacetylgalactosamine- Product Code: BMN 110 INN or Proposed INN: Not Available Other descriptive name: recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 162 | Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Turkey;Colombia;Italy;Switzerland;United Kingdom;France;Canada;Argentina;Brazil;Poland;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of |