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 | NCT01976091 (ClinicalTrials.gov) | February 1, 2015 | 24/7/2013 | A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D) | Phase I/IIA Gene Transfer Clinical Trial for LGMD2D (Alpha-Sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA | Limb-Girdle Muscular Dystrophy, Type 2D | Genetic: SRP-9004 | Sarepta Therapeutics, Inc. | Nationwide Children's Hospital | Completed | 7 Years | N/A | All | 6 | Phase 1/Phase 2 | United States |