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	EUCTR2006-000136-27-DE (EUCTR)	17/03/2008	04/09/2007	Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children with Urea Cycle Disorders (UCDs)	Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children with Urea Cycle Disorders (UCDs)	Neonates and Infants up to 3 months including with prenatally or postnatally confirmed urea cycle disorder with below listed deficiency and children aged > 3 months up to 5 years including with confirmed urea cycle disorder and unstable metabolism with deficiency of either: - Carbamyl-phosphate Synthetase I Deficiency (CPS1D) - Ornithine Transcarbamylase Deficiency (OTCD) - Argininosuccinate Synthetase Deficiency (ASSD/Citrullinaemia)can be included. MedDRA version: 18.1;Level: PT;Classification code 10052450;Term: Ornithine transcarbamoylase deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10058298;Term: Argininosuccinate synthetase deficiency;Classification code 10058297;Term: Carbamoyl phosphate synthetase deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: Human heterologous liver cells (for infusion) Product Code: HHLivC	Cytonet GmbH & Co KG	NULL	Not Recruiting			Female: yes Male: yes			Germany

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EUCTR2006-000136-27-DE
(EUCTR)

17/03/2008

04/09/2007

Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children with Urea Cycle Disorders (UCDs)

Neonates and Infants up to 3 months including with prenatally or postnatally confirmed urea cycle disorder with below listed deficiency and children aged > 3 months up to 5 years including with confirmed urea cycle disorder and unstable metabolism with deficiency of either: - Carbamyl-phosphate Synthetase I Deficiency (CPS1D) - Ornithine Transcarbamylase Deficiency (OTCD) - Argininosuccinate Synthetase Deficiency (ASSD/Citrullinaemia)can be included.
MedDRA version: 18.1;Level: PT;Classification code 10052450;Term: Ornithine transcarbamoylase deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10058298;Term: Argininosuccinate synthetase deficiency;Classification code 10058297;Term: Carbamoyl phosphate synthetase deficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: Human heterologous liver cells (for infusion)
Product Code: HHLivC

Cytonet GmbH & Co KG

NULL

Not Recruiting

Female: yes
Male: yes

Germany