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 | NCT04713475 (ClinicalTrials.gov) | March 17, 2021 | 4/1/2021 | Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 Gangliosidosis | Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Subjects With Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis | GM1 Gangliosidosis;GM1 Gangliosidosis, Type I;GM1 Gangliosidosis, Type 2;Beta-Galactosidase-1 (GLB1) Deficiency | Biological: PBGM01 | Passage Bio, Inc. | NULL | Active, not recruiting | 4 Months | 36 Months | All | 20 | Phase 1/Phase 2 | United States;Brazil;Canada;Turkey;United Kingdom |