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	NCT02670889 (ClinicalTrials.gov)	November 2016	14/1/2016	Urease Inhibitor Drug Treatment for Urea Cycle Disorders	Manipulating the Gut Microbiome in Urea Cycle Disorders	Ornithine Transcarbamylase Deficiency;Argininosuccinate Synthetase Deficiency (Citrullinemia);Argininosuccinic Acid Lyase Deficiency (Argininosuccinic Aciduria);Carbamyl-Phosphate Synthase I Deficiency	Drug: Acetohydroxamic Acid;Drug: Isotopic Intravenous [13C]-Urea	Nicholas Ah Mew	Data Management and Coordinating Center (DMCC);Children's Hospital of Philadelphia	Not yet recruiting	18 Years	60 Years	Both	16	Phase 1;Phase 2	NULL

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

NCT02670889
(ClinicalTrials.gov)

November 2016

14/1/2016

Urease Inhibitor Drug Treatment for Urea Cycle Disorders

Manipulating the Gut Microbiome in Urea Cycle Disorders

Ornithine Transcarbamylase Deficiency;Argininosuccinate Synthetase Deficiency (Citrullinemia);Argininosuccinic Acid Lyase Deficiency (Argininosuccinic Aciduria);Carbamyl-Phosphate Synthase I Deficiency

Drug: Acetohydroxamic Acid;Drug: Isotopic Intravenous [13C]-Urea

Nicholas Ah Mew

Data Management and Coordinating Center (DMCC);Children's Hospital of Philadelphia

Not yet recruiting

18 Years

60 Years

Both

Phase 1;Phase 2

NULL