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 | NCT04240314 (ClinicalTrials.gov) | January 15, 2020 | 22/1/2020 | AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. | Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: scAAV9.U7.ACCA | Megan Waldrop | Audentes Therapeutics | Active, not recruiting | 6 Months | 13 Years | Male | 3 | Phase 1/Phase 2 | United States |