A clinical trial of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 2
Phase 2
- Conditions
- progressive familial intrahepatic cholestasis type 2
Recruitment & Eligibility
- Status
- Complete: follow-up continuing
- Sex
- All
- Target Recruitment
- 6
Inclusion Criteria
Not provided
Exclusion Criteria
1)Those who participated in another study within 3 months 2)Those who have hypersensitivity with phenylbutylate and/or its metabolite 3)Those who received liver transplantation 4)Those who received biliary diversion 5)Those who are pregnant or hoping pregnancy 6)Those who cannot accept to practice contraception 7)Those with congestive heart failure 8)Those with renal dysfunction 9)Those with nonsense or frameshift mutation, or exon deletion in both ABCB11 alleles 10) Those with liver cirrhosis
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To evaluate liver histopathology before and after 24-weeks treatment with sodium phenylbutylate
- Secondary Outcome Measures
Name Time Method 1)Liver histopathological score 2)Reliability of histopathological scoring system 3)Biomarkers such as AST, ALT, g-GTP, T.Bil, D.Bil 4)Itching 5)Biomarkers such as plasma amino acid fraction (isoleucine, glutamine, glutamic acid, argineine), and anmonia 6)Survival rate 7)Liver transplantation avoidance rate 8)Pharmacokinetics 9)Adverse events