113. Muscular dystrophy Clinical trials / Disease details
Clinical trials : 622 / Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168
| 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 | NCT01957059 (ClinicalTrials.gov) | June 2013 | 2/7/2013 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy | Drug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing Extension | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | 18 Years | Male | 9 | Phase 1/Phase 2 | Belgium;France;Italy;Netherlands;United Kingdom |