MRT5201 ( 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 : 48 薬物数 : 52 - (DrugBank : 13) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 29
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03767270
(ClinicalTrials.gov)
December 201928/11/2018Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC DeficiencyA Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenously Administered MRT5201 in Subjects With Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase DeficiencyBiological: MRT5201;Other: PlaceboTranslate Bio, Inc.NULLWithdrawn18 YearsN/AAll0Phase 1/Phase 2NULL