Unique ID issued by UMIN | UMIN000008528 |
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Receipt number | R000010027 |
Scientific Title | Sodium pyruvate therapy for the patients with citrin deficiency |
Date of disclosure of the study information | 2012/09/15 |
Last modified on | 2018/08/27 11:52:13 |
Sodium pyruvate therapy for the patients with citrin deficiency
pyruvate therapy for citrin deficiency
Sodium pyruvate therapy for the patients with citrin deficiency
pyruvate therapy for citrin deficiency
Japan |
citrin deficiency
Hepato-biliary-pancreatic medicine | Neurology | Pediatrics |
Others
NO
We examinie effects of sodium pyruvate on neonatal intrahepatic cholestasis caused by citrin deficiency(NICCD), easy fatigability and hypoglycemia in the compensation period and adult onset type II citrullinemia (CTLN2) in citrin deficiency.
Efficacy
Adult onset type II citrullinemia
1) improvement of citrulllinemia and hyperannmonemia
2) improvement of impaired consciousness
3) improvement of QOL
Compensation period
1) improvement of citrulllinemia and oxidative stress
2) improvement of QOL
3) improvement of appetite habit
Neonatal intrehepatic cholestasis caused by citrin deficincy
1) improvement of cholestasis and citrullinemia
2) catch up of body weight
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
1
Treatment
Medicine |
1) Medicine: sodium pyruvate
2) Duration: 16 weeks, If patient hopes, it can continue.
3) Amount: 100~300 mg/kg/day
4) Number:2~3 times/day after eating orally
Not applicable |
Not applicable |
Male and Female
1) the patients with CTLN2
2) the patients with symptoms of easy fatiqability growth retardation and hypoglycemia in compensation period.
3) the patients with severe hepatic damage by NICCD.
4) the patients with informed concecnt by document was proved.
pregnant woman
6
1st name | |
Middle name | |
Last name | Yasuhiro Takeshima |
Kobe University Graduate School of Medicine
Department of Pediatrics
7-5-1, Kusunoki-cho, Chuo, Kobe, 6500017, Japan
078-382-5111
takesima@med.kobe-u.ac.jp
1st name | |
Middle name | |
Last name | Hiroyuki AWANO |
Kobe University Graduate School of Medicine
Department of Pediatrics
7-5-1, Kusunoki-cho, Chuo, Kobe, 6500017, Japan
078-382-5111
awahiro@med.kobe-u.ac.jp
Department of Pediatrics, Kobe university Graduate School of Medicine
Grant-in-Aid for Scientific Research
NO
2012 | Year | 09 | Month | 15 | Day |
Unpublished
Completed
2012 | Year | 09 | Month | 15 | Day |
2012 | Year | 09 | Month | 15 | Day |
2012 | Year | 07 | Month | 25 | Day |
2018 | Year | 08 | Month | 27 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000010027
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