DDrare: Database of Drug Development for Rare Diseases

Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene (DrugBank: Ornithine)

2 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
245	プロピオン酸血症	0
251	尿素サイクル異常症	1

245. プロピオン酸血症　［臨床試験数：9，薬物数：15（DrugBank：3），標的遺伝子数：1，標的パスウェイ数：6］

Searched query = "Propionic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 9 trial found

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.

1 / 42 trial found

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	NCT00004386 (ClinicalTrials.gov)	October 1995	18/10/1999	Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency		Ornithine Transcarbamylase Deficiency Disease	Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Children's Research Institute	Terminated	18 Years	65 Years	Both		Phase 1	NULL