251. 尿素サイクル異常症
[臨床試験数:42,薬物数:45(DrugBank:10),標的遺伝子数:4,標的パスウェイ数:29]
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.
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 | NCT04442347 (ClinicalTrials.gov) | October 2020 | 19/6/2020 | Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency | A 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 Deficiency | Ornithine Transcarbamylase Deficiency | Biological: ARCT-810;Other: Placebo | Arcturus Therapeutics, Inc. | NULL | Recruiting | 18 Years | N/A | All | 12 | Phase 1 | United States |
2 | NCT04416126 (ClinicalTrials.gov) | June 1, 2020 | 28/5/2020 | Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects | A 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 Subjects | Ornithine Transcarbamylase Deficiency | Biological: ARCT-810;Other: Placebo | Arcturus Therapeutics, Inc. | NULL | Recruiting | 18 Years | 65 Years | All | 30 | Phase 1 | New Zealand |