19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | NCT02294877 (ClinicalTrials.gov) | September 2014 | 28/10/2014 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | Mucopolysaccharidosis IV Type A;Morquio A Syndrome;MPS IVA | Drug: Vimizim® (elosulfase alfa) | BioMarin Pharmaceutical | ICON plc | Active, not recruiting | N/A | N/A | All | 425 | United States;Australia;Austria;Belgium;Canada;Czechia;Denmark;France;Ireland;Italy;Malaysia;Netherlands;Poland;Portugal;Puerto Rico;Taiwan;United Kingdom;Germany |