307. カナバン病 Canavan disease Clinical trials / Disease details
臨床試験数 : 5 / 薬物数 : 9 - (DrugBank : 3) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2
| 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 | NCT04833907 (ClinicalTrials.gov) | April 1, 2021 | 24/3/2021 | rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease | Phase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of AVASPA Gene Therapy for Treatment of Children With Typical Canavan Disease | Canavan Disease | Drug: rAAV-Olig001-ASPA;Drug: Levetiracetam;Drug: Prednisolone | Myrtelle Inc. | NULL | Recruiting | 3 Months | 60 Months | All | 24 | Phase 1/Phase 2 | United States |