19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 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 | NCT01415427 (ClinicalTrials.gov) | July 2011 | 8/8/2011 | Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (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) | Mucopolysaccharidosis IV A;Morquio A Syndrome;MPS IVA | Drug: BMN 110 - Weekly;Drug: BMN 110 - Every Other Week | BioMarin Pharmaceutical | NULL | Completed | 5 Years | N/A | All | 173 | Phase 3 | United States;Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom |