Bmn 053 (DrugBank: -)
1 disease| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 113 | 筋ジストロフィー | 1 |
| 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 | EUCTR2011-005042-35-GB (EUCTR) | 23/08/2013 | 18/03/2013 | A study to test if BMN 053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by BMN 053-induced DMD exon 53 skipping MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 1;Phase 2 | United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan |