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 | NCT04637282 (ClinicalTrials.gov) | September 1, 2021 | 4/11/2020 | Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3 | A Randomized. Multicenter, Double-Blind, Placebo-Controlled Safety, Tolerability, and Efficacy Study of PLX-200 in Participants With Mild-to-Moderate Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Disease | Juvenile Neuronal Ceroid Lipofuscinosis | Drug: PLX-200;Drug: Placebo | Polaryx Therapeutics, Inc. | NULL | Not yet recruiting | 6 Years | 18 Years | All | 39 | Phase 3 | NULL |