AAVLK03hOTC ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
251尿素サイクル異常症1

251. 尿素サイクル異常症


臨床試験数 : 54 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05092685
(ClinicalTrials.gov)
February 1, 202228/9/2021Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEnPhase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for Paediatric Patients With Ornithine Transcarbamylase Deficiency.Ornithine Transcarbamylase DeficiencyGenetic: AAVLK03hOTCUniversity College, LondonNULLNot yet recruitingN/A16 YearsAll12Phase 1/Phase 2NULL