Clinical study of HAES transplantation in patients with neonatal onset urea cycle disorder

General Information
Summary	HAES is derived from human ES cells and directed to differentiate to cells with ammonia metabolism capacity. HAES transplantation is expected as an effective bridging therapy to liver transplantation for patients with urea cycle disorder. This study is conducted to assess the safety and efficacy of HAES transplantation for the neonatal onset patients who have difficulty in instant liver transplantation surgery due to low body weight (6 kg or less).
Clinical trials phase	Phases 1/2
Start date (estimated)	2018-04-30
End date (estimated)	2022-09-30
Clinical feature	Label urea cycle disorder Link http://purl.obolibrary.org/obo/DOID_9267 Description An amino acid metabolic disorder that involves a deficiency of one of the enzymes in the urea cycle which is responsible for removing ammonia from the blood stream.
Administrative Information
ICTRP weblink	https://trialsearch.who.int/Trial2.aspx?TrialID=JPRN-JMA-IIA00412
Other study identifiers	Name JMA-IIA00412 Description J​M​A​C​C​T​ ​I​D
Source weblink	https://dbcentre3.jmacct.med.or.jp/jmactr/App/JMACTRE02_04/JMACTRE02_04.aspx?kbn=3&seqno=9141
Public contact	Email f​u​k​u​h​a​r​a​-​y​s​@​n​c​c​h​d​.​g​o​.​j​p Public email f​u​k​u​h​a​r​a​-​y​s​@​n​c​c​h​d​.​g​o​.​j​p First name Y​a​s​u​y​u​k​i Last name F​u​k​u​h​a​r​a Phone +81-3-3416-0181 Fax +​8​1​-​3​-​3​4​1​6​-​2​2​2​2 City Tokyo Country Japan Address freetext 2​-​1​0​-​1​ ​O​k​u​r​a​ ​S​e​t​a​g​a​y​a​ ​T​o​k​y​o​ ​1​5​7​-​8​5​3​5
Sponsors	National center for child health and development
Cells
Which differentiated cell type is used	Label hepatocyte Link http://purl.obolibrary.org/obo/CL_0000182 Description The main structural component of the liver. They are specialized epithelial cells that are organized into interconnected plates called lobules. Majority of cell population of liver, polygonal in shape, arranged in plates or trabeculae between sinusoids; may have single nucleus or binucleated.; Hepatocytes are reportedly MHC Class I-positive and MHC Class II-positive.
Recruitment
Recruitment Status	Recruiting
Comment recruitment status	Performed number of subjects: 2
Estimated number of participants	5