257. 肝型糖原病 Hepatic glycogenosis Clinical trials / Disease details
臨床試験数 : 14 / 薬物数 : 28 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 8
| 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 | NCT04990388 (ClinicalTrials.gov) | October 18, 2021 | 26/7/2021 | Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) | A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients With GSD III | Glycogen Storage Disease Type III | Biological: UX053;Other: Placebo;Drug: Antipyretic;Drug: H2 Blocker;Drug: H1 Blocker | Ultragenyx Pharmaceutical Inc | NULL | Recruiting | 18 Years | N/A | All | 30 | Phase 1/Phase 2 | United States;Canada;France;Italy;Spain;United Kingdom |