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

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000012782

Enrollment

Registered

2014-02-01

Start date

2014-02-01

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

Group A

phenylbutyrate 6g (Child 100mg/kg)/day *7days Group B

phenylbutyrate 6g (Child 100mg/kg)/day *3days and 12g (Child 200mg/kg)/day *4days Group C

phenylbutyrate 6g (Child 100mg/kg)/day *1day, phenylbutyrate 12g (Child 200mg/kg)/day *2days and phenylbutyrate 21g (Child 300mg/kg)/day *4days

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 ContactNakayuki Naritaka

Juntendo University Department of Pediatrics and Adolescent Medicine

naritaka@juntendo.ac.jp+81-(0)3-3813-3111(3325)

Outcome results

None listed

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