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-IT (EUCTR) | 20/12/2011 | 27/12/2011 | A phase 3 extension study to evaluate the long term efficacy and safetyof BMN 110 in patients with Mucopolysaccharidosis IV A (Morquio Asyndrome) | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with MucopolysaccharidosisIVA (Morquio A Syndrome) | 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: BM110 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;Korea, Democratic People's Republic of;Turkey;Colombia;United Kingdom;Italy;Switzerland;Canada;Argentina;Brazil;Denmark;Australia;Germany;Japan |