Antipyretic ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
257肝型糖原病1

257. 肝型糖原病


臨床試験数 : 14 薬物数 : 27 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 8
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04990388
(ClinicalTrials.gov)
October 18, 202126/7/2021Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD IIIGlycogen Storage Disease Type IIIBiological: UX053;Other: Placebo;Drug: Antipyretic;Drug: H2 Blocker;Drug: H1 BlockerUltragenyx Pharmaceutical IncNULLActive, not recruiting18 YearsN/AAll8Phase 1/Phase 2United States;Canada;France;Italy;Spain;United Kingdom