19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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 | NCT04669535 (ClinicalTrials.gov) | January 15, 2021 | 23/11/2020 | A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease | A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease | Tay-Sachs Disease;Sandhoff Disease | Biological: AXO-AAV-GM2 Starting Dose;Biological: AXO-AAV-GM2 Low Dose;Biological: AXO-AAV-GM2 Middle Dose;Biological: AXO-AAV-GM2 High Dose | Sio Gene Therapies | Massachusetts General Hospital;University of Massachusetts, Worcester | Recruiting | 6 Months | 12 Years | All | 18 | Phase 1 | United States |