113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
| 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 | NCT04226924 (ClinicalTrials.gov) | June 15, 2017 | 15/7/2019 | Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose | A Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment of | Oculopharyngeal Muscular Dystrophy | Drug: Trehalose | Bioblast Pharma Ltd. | NULL | Withdrawn | 50 Years | 70 Years | All | 0 | Phase 2 | Canada |
| 2 | NCT02328482 (ClinicalTrials.gov) | January 2015 | 25/12/2014 | Continuation Protocol to Protocol BBCO-001 | A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in OPMD Patients Who Participated in Study BBCO-001 | Muscular Dystrophy, Oculopharyngeal (OPMD) | Drug: Tehalose 30gr | Bioblast Pharma Ltd. | NULL | Completed | 18 Years | 80 Years | All | 9 | Phase 3 | Canada |
| 3 | NCT02015481 (ClinicalTrials.gov) | February 2014 | 8/12/2013 | Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients | Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients | Oculopharyngeal Muscular Dystrophy | Drug: Cabaletta | Bioblast Pharma Ltd. | NULL | Completed | 18 Years | 80 Years | All | 25 | Phase 2 | United States;Canada;Israel |