Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-UMIN000003802
• Date of registration: 22/06/2010
• Prospective Registration: No
• Primary sponsor: Saiseikai Yokohama City Tobu Hospital
• Public title: Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.
• Scientific title: Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome. - Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.
• Date of first enrolment: 2010/04/01
• Target sample size: 15
• Recruitment status: Recruiting
• URL: https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000004376
• Study type: Interventional
• Study design: Single arm Non-randomized
• Phase: Not selected

Countries of recruitment

Japan

Contacts

• Name: Ayano Inui
• Affiliation: Saiseikai Yokohama City Tobu Hospital Division of Hepatology and Gastroenterology, Children's Center for Health and Development

• Name: Ayano Inui
• Affiliation: Saiseikai Yokohama City Tobu Hospital Division of Hepatology and Gastroenterology, Children's Center for Health and Development

Key inclusion & exclusion criteria

Inclusion criteria:
Exclusion criteria: History of drug hypersensitivity Severe liver injury Renal function impairment Severe complication Poor medical compliance

Age minimum: Not applicable
Age maximum: Not applicable
Gender: Male and Female

Health Condition(s) or Problem(s) studied

Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.

Intervention(s)

phenylbutyrate(Child 250mg/kg/day)for 1-4months : phenylbutyrate(Child 350mg/kg/day)for 1-4months : phenylbutyrate(Child 500mg/kg/day)for 1-4months

Primary Outcome(s)

subjective and objective symptom (pruritus, jaundice) T-Bil, D-Bil, AST, ALT, LDH, ALP, TBA, urine pH, glucose, occult blood, bilirubin, urobilinogen.

Source(s) of Monetary Support

MEXT scientific research fund

Secondary Sponsor(s)

Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo (Tokyo)

Ethics review

Status: YES
Approval date:
Contact: