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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
3 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03064048September 15, 201710/2/2017Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLDEffect of Nitric Oxide (NO) Supplementation on Neurocognitive Measures in Argininosuccinate Lyase Deficiency (ASLD)Argininosuccinate Lyase Deficiency;Urea Cycle Disorder;Urea Cycle Disorders, Inborn;Argininosuccinic AciduriaDietary Supplement: Neo-ASA;Dietary Supplement: PlaceboBaylor College of MedicineRare Diseases Clinical Research Network;Neogenis LaboratoriesRecruiting6 Years50 YearsAll16N/AUnited States
2NCT02252770September 201425/9/2014Nitric Oxide Supplementation in Argininosuccinic AciduriaNitric Oxide Supplementation as a Therapeutic Intervention in Argininosuccinic AciduriaArgininosuccinic Aciduria;Argininosuccinate Lyase Deficiency;Urea Cycle DisordersDietary Supplement: Nitric oxide supplement;Dietary Supplement: PlaceboBaylor College of MedicineNULLCompleted8 Years64 YearsAll12N/AUnited States
3NCT00345605February 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