251. Urea cycle disorder
42 clinical trials,   45 drugs   (DrugBank: 10 drugs),   4 drug target genes,   29 drug target pathways

Searched query = "Urea cycle disorder", "N-acetylglutamate synthase deficiency", "NAGS deficiency", "Carbamoyl phosphate synthetase I deficiency", "CPS1 deficiency", "Ornithine transcarbamylase deficiency", "OTC deficiency", "Classic citrullinemia", "Citrullinemia type I", "Argininosuccinic aciduria", "Argininemia", "Hiperornitinemia-hiperamonemia-homocitrulinuria syndrome", "HHH syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
1 trial found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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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