113. 筋ジストロフィー
[臨床試験数:567,薬物数:442(DrugBank:93),標的遺伝子数:55,標的パスウェイ数:151]
Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
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 | NCT02329769 (ClinicalTrials.gov) | December 2014 | 22/12/2014 | Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: PRO044 SC 6 mg/kg;Drug: PRO044 IV 6 mg/kg;Drug: PRO044 IV 9 mg/kg | BioMarin Pharmaceutical | NULL | Terminated | 9 Years | 20 Years | Male | 15 | Phase 2 | Belgium;Italy;Netherlands;Sweden |
2 | NCT01037309 (ClinicalTrials.gov) | December 2009 | 21/12/2009 | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | A Phase I/IIa, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous and Intravenous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: PRO044 SC;Drug: PRO044 IV | BioMarin Pharmaceutical | NULL | Completed | 5 Years | 16 Years | Male | 18 | Phase 1;Phase 2 | Belgium;Italy;Netherlands;Sweden |