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 |
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1 | EUCTR2008-003865-23-GB (EUCTR) | 08/09/2008 | 25/07/2008 | A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle Disorders | A Phase 2, Switch-Over, Dose-Escalation Study of the Safety and Tolerability of HPN-100 (glyceryl tri-[4-phenylbutyrate]) Compared to Sodium Phenylbutyrate in Adult and Pediatric Subjects with Urea Cycle Disorders | Urea Cycle Disorder (urea cycle enzyme or transporter deficiency) MedDRA version: 9.1;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism | Product Name: glyceryl tri-(4-phenylbutyrate)(GT4P) Product Code: HPN-100 INN or Proposed INN: NA Other descriptive name: glyceryl tri-(4-phenylbutyrate) | Hyperion Therapeutics Limited | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United Kingdom |