臨床研究等提出・公開システム

Scientific Title	Confirmation of effects of medium chain triglyceride therapy for citrin deficiency, especially in patients with symptomatic post neonatal intrahepatic cholestasis and that with adult-onset type II citrullinemia
Public Title	Confirmation of effects of medium chain triglyceride therapy for citrin deficiency

Contact for Scientific Queries	Name	Mitsui Tetsuo
	Affiliation	Yamagata university faculty of medicine
	Address	2-2-2, Iida-nishi, Yamagata-city, Yamagata, 990-9585, JAPAN
	Telephone	+81-23-633-1122
	E-mail	tmitsui@med.id.yamagata-u.ac.jp
Contact for Public Queries	Name	Numakura Chikahiko
	Affiliation	Yamagata University Hospital
	Address	2-2-2, Iida-nishi, Yamagata-city, Yamagata, 990-9585, JAPAN
	Telephone	+81-23-628-5329
	E-mail	cnumakur@med.id.yamagata-u.ac.jp

Recruitment status	Recruiting

Anticipated date of first enrollment		July. 31, 2012
Actual date of first enrollment		July. 31, 2012
Target sample size		50
Study Type		Interventional
Study Design	allocation	single arm study
	masking	open(masking not used)
	control	no treatment control/standard of care control
	assignment	single assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	1) Patients with symptomatic post NICCD 2) Patients with adult-onset type II citrullinemia 3) Patients with biallelic mutation of SLC25A13 gene
	Exclusion Criteria	1) Patients with sodium pyruvate therapy 2) liver transplanted patients 3) Judged inappropriate for this study by the physicians.
	Age Minimum	0age
	Age Maximum	80age old
	Gender	Both
Health Condition(s) or Problem(s) Studied		citrin deficiency
Intervention(s)		Medium chain triglyceride 5-15ml 3 times a day
Health Condition(s) Keyword		Endocrinology and Metabolism, Pediatrics
Intervention(s) Keyword
Health Condition(s) Code		D006111
Intervention(s) Code
Primary Outcome(s)		Improvemet of clinical symptoms and QOL
Secondary Outcome(s)		1) plasma ammonia concentration 2) plasma citrulline concentration 3) plasma Fisher ratio 4) auxological data

Primary Sponsor

Primary Sponsor

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support	AMED (Japan)
Secondary Sponsor	Not applicable

Name of Certified Review Board	Institutional Review Board, Shizuoka Cancer Center
Address	1007 Shimonagakubo, Nagaizumi-cho, Sunto-gun, Shizuoka Prefecture 411-8777 Japan, Shizuoka
Telephone	+81-55-989-5222
E-Mail	rinsho_office@scchr.jp
Approval Status	Approval
Date of approval	Mar. 08, 2019

Plan to share IPD
Plan description

Secondary ID(s)
Issuing Authority

Countries of Recruitment（Except Japan）	none