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 | NCT05629559 (ClinicalTrials.gov) | October 16, 2022 | 4/11/2022 | 4D-310 in Adults With Fabry Disease and Cardiac Involvement | An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement | Fabry Disease | Biological: 4D-310 | 4D Molecular Therapeutics | NULL | Active, not recruiting | 18 Years | N/A | All | 18 | Phase 1/Phase 2 | Australia;Taiwan |
2 | NCT04519749 (ClinicalTrials.gov) | September 1, 2020 | 14/8/2020 | An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease | An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease | Fabry Disease | Biological: 4D-310 | 4D Molecular Therapeutics | NULL | Active, not recruiting | 18 Years | N/A | All | 18 | Phase 1/Phase 2 | United States |