251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details


臨床試験数 : 54 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28

  
3 trials found
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1NCT03335488
(ClinicalTrials.gov)
February 20, 201818/10/2017Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle DisordersA 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 DisordersUrea Cycle DisorderDrug: RAVICTI;Drug: NaPBAHorizon Therapeutics, LLCNULLCompletedN/A99 YearsAll16Phase 4United States;Italy;Spain;Switzerland;Belgium
2EUCTR2008-003865-23-GB
(EUCTR)
08/09/200825/07/2008A 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 DisordersA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
16Phase 2United Kingdom
3NCT00345605
(ClinicalTrials.gov)
February 200826/6/2006Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle DisorderA Randomized, Double-Blind, Crossover Study of Sodium Phenylbutyrate and Low-Dose Arginine Compared to High-Dose Arginine Alone on Liver Function, Ureagenesis and Subsequent Nitric Oxide Production in Patients With Argininosuccinic AciduriaArgininosuccinic Aciduria;Amino Acid Metabolism, Inborn Errors;Urea Cycle DisordersDrug: Sodium Phenylbutyrate;Drug: ArginineBrendan LeeOffice of Rare Diseases (ORD);Rare Diseases Clinical Research Network;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Completed5 YearsN/AAll12Phase 2United States