19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
| 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 | NCT05324943 (ClinicalTrials.gov) | April 15, 2022 | 28/2/2022 | A Gene Therapy Study in Patients With Gaucher Disease Type 1 | A Phase 1/2, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1) | Gaucher Disease, Type 1 | Genetic: FLT201 | Freeline Therapeutics | NULL | Recruiting | 18 Years | N/A | All | 18 | Phase 1/Phase 2 | Argentina;Brazil;Germany;Israel;Paraguay;Spain;United Kingdom;Italy;Tunisia |