DDrare: Database of Drug Development for Rare Diseases

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.

Drug = 4-phenylbutyrate; ALLOGENIC HUMAN HETEROLOGOUS LIVER CELLS; ARCT-810; ASA; Acetohydroxamic Acid; Acetohydroxamic Acid Oral Tablet [Lithostat]; Acetohydroxamic acid; Arginine; Behavioral: Protein and calorie controlled diet; Carglumic acid; Celulas hepatocitos troncales adultas alogenicas de higado expandidas; DTX301; GT4P; Genetic: Ornithine transcarbamylase vector; Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene; Genetic: scAAV8OTC; Glyceryl tri-(4-phenylbutyrate); Glyceryl tri-(4-phenylbutyrate)(GT4P); HHALPC; HHLivC; HPN-100; HepaStem; HepaStem Infusion; Heparesc; Heterologous Human Adult Liver-derived Progenitor Cells; Heterologous Human Adult Liver-derived Progenitor Cells (HHALPC); Human Heterologous Liver Cells; Human heterologous liver cells (for infusion); Isotopic Intravenous [13C]-Urea; KB195; MRT5201; NaPBA; Neo-ASA; Nitric Oxide; Nitric oxide supplement; Oral prednisone; Ornithine; Phenylbutyrate; Prednisone; Promethera HepaStem; RAVICTI; Ravicti; SYNB1020; Sodium Phenylbutyrate; Urea

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	NCT03064048 (ClinicalTrials.gov)	September 15, 2017	10/2/2017	Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD	Effect of Nitric Oxide (NO) Supplementation on Neurocognitive Measures in Argininosuccinate Lyase Deficiency (ASLD)	Argininosuccinate Lyase Deficiency;Urea Cycle Disorder;Urea Cycle Disorders, Inborn;Argininosuccinic Aciduria	Dietary Supplement: Neo-ASA;Dietary Supplement: Placebo	Baylor College of Medicine	Rare Diseases Clinical Research Network;Neogenis Laboratories	Recruiting	6 Years	50 Years	All	16	N/A	United States
2	NCT02252770 (ClinicalTrials.gov)	September 2014	25/9/2014	Nitric Oxide Supplementation in Argininosuccinic Aciduria	Nitric Oxide Supplementation as a Therapeutic Intervention in Argininosuccinic Aciduria	Argininosuccinic Aciduria;Argininosuccinate Lyase Deficiency;Urea Cycle Disorders	Dietary Supplement: Nitric oxide supplement;Dietary Supplement: Placebo	Baylor College of Medicine	NULL	Completed	8 Years	64 Years	All	12	N/A	United States
3	NCT00345605 (ClinicalTrials.gov)	February 2008	26/6/2006	Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder	A 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 Aciduria	Argininosuccinic Aciduria;Amino Acid Metabolism, Inborn Errors;Urea Cycle Disorders	Drug: Sodium Phenylbutyrate;Drug: Arginine	Brendan Lee	Office of Rare Diseases (ORD);Rare Diseases Clinical Research Network;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Completed	5 Years	N/A	All	12	Phase 2	United States

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

NCT03064048
(ClinicalTrials.gov)

September 15, 2017

10/2/2017

Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

Effect of Nitric Oxide (NO) Supplementation on Neurocognitive Measures in Argininosuccinate Lyase Deficiency (ASLD)

Argininosuccinate Lyase Deficiency;Urea Cycle Disorder;Urea Cycle Disorders, Inborn;Argininosuccinic Aciduria

Dietary Supplement: Neo-ASA;Dietary Supplement: Placebo

Baylor College of Medicine

Rare Diseases Clinical Research Network;Neogenis Laboratories

Recruiting

6 Years

50 Years

All

N/A

United States

NCT02252770
(ClinicalTrials.gov)

September 2014

25/9/2014

Nitric Oxide Supplementation in Argininosuccinic Aciduria

Nitric Oxide Supplementation as a Therapeutic Intervention in Argininosuccinic Aciduria

Argininosuccinic Aciduria;Argininosuccinate Lyase Deficiency;Urea Cycle Disorders

Dietary Supplement: Nitric oxide supplement;Dietary Supplement: Placebo

Baylor College of Medicine

NULL

Completed

8 Years

64 Years

All

N/A

United States

NCT00345605
(ClinicalTrials.gov)

February 2008

26/6/2006

Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder

A 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 Aciduria

Argininosuccinic Aciduria;Amino Acid Metabolism, Inborn Errors;Urea Cycle Disorders

Drug: Sodium Phenylbutyrate;Drug: Arginine

Brendan Lee

Office of Rare Diseases (ORD);Rare Diseases Clinical Research Network;Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Completed

5 Years

N/A

All

Phase 2

United States