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

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000003802

Enrollment

Registered

2010-06-22

Start date

2010-04-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

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

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients with caretaker's approval

Exclusion criteria

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

Design outcomes

Primary

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

Countries

Japan

Contacts

Public ContactAyano Inui

Saiseikai Yokohama City Tobu Hospital Division of Hepatology and Gastroenterology, Children's Center for Health and Development

a_murayama@yobu.saiseikai.or.jp(045)576-3000

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026