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 |
JMACCT ID |
|
Source weblink |
https://dbcentre3.jmacct.med.or.jp/jmactr/App/JMACTRE02_04/JMACTRE02_04.aspx?kbn=3&seqno=9141 |
Public contact |
Email |
fukuhara-ys@ncchd.go.jp |
Public email |
fukuhara-ys@ncchd.go.jp |
First name |
Yasuyuki |
Last name |
Fukuhara |
Phone |
+81-3-3416-0181 |
Fax |
+81-3-3416-2222 |
City |
Tokyo |
Country |
|
Address freetext |
2-10-1 Okura Setagaya Tokyo 157-8535 |
|
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 |