Refractory pediatric cholestasis disease (progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis, Alagille syndrome, drug-induced cholestatic liver injury, biliary atresia, etc)
Conditions
Sponsors
Juntendo University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Subjects with diagnosis of refractory pediatric cholestasis disease. 2) Hospitalised patients. 3) Those who can intake medicine orally. 4) Those who can receive the observation essential for this study. 5) Subjects who have been determined eligible by principal investigator or sub-investigator from physical and clinical examinations. 6) Those who have agreed with participating in this study by written informed consent prior to the study (patients or legally acceptable representative).
Exclusion criteria
Exclusion criteria: 1) Those who have hypersensitivity with phenylbutylate, its metabolites, drug additives and/or benzoate, etc 2) Subjects with congestive heart failure, kidney failure, and/or hypernatremia with edema 3) Subjects with renal dysfunction 4) Subjects with liver cirrhosis 5) Subjects who have been determined ineligible by principal investigator or sub-investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To determine concentration of phenylbutylate and its metabolites in blood and urine after oral administration of sodium phenylbutyrate | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Plasma amino acid fraction (isoleucine, glutamine, glutamic acid, argineine), and anmonia 2) Adverse events | — |
Countries
Japan
Contacts
Public ContactKei Minowa
Juntendo University, Faculty of Medicine Department of Pediatrics
Outcome results
None listed