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.
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 | NCT03335488 (ClinicalTrials.gov) | February 20, 2018 | 18/10/2017 | Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders | A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders | Urea Cycle Disorder | Drug: RAVICTI;Drug: NaPBA | Horizon Therapeutics, LLC | NULL | Recruiting | N/A | 99 Years | All | 18 | Phase 4 | United States;Belgium;Italy;Spain;Switzerland |
2 | NCT02246218 (ClinicalTrials.gov) | December 31, 2014 | 17/9/2014 | A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders | An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs) | Urea Cycle Disorder | Drug: RAVICTI | Horizon Therapeutics, LLC | NULL | Completed | N/A | 2 Years | All | 27 | Phase 4 | United States;Canada |
3 | 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 |