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"
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT04442347
(ClinicalTrials.gov)
October 202019/6/2020Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase DeficiencyA Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients With Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase DeficiencyBiological: ARCT-810;Other: PlaceboArcturus Therapeutics, Inc.NULLRecruiting18 YearsN/AAll12Phase 1United States
2NCT04416126
(ClinicalTrials.gov)
June 1, 202028/5/2020Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult SubjectsA Phase 1 Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Healthy Adult SubjectsOrnithine Transcarbamylase DeficiencyBiological: ARCT-810;Other: PlaceboArcturus Therapeutics, Inc.NULLRecruiting18 Years65 YearsAll30Phase 1New Zealand