An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1

An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1 - An exploratory study of efficacy and safety of sodium phenylbutyrate in progressive familial intrahepatic cholestasis type 1

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000027666

Enrollment

Registered

2017-07-01

Start date

2017-07-01

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 type 1

Interventions

To subscribe 450 or 600mg/kg/day sodium phenylbutylate (buphenyl) for 12 months

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1)Patients who are diagnosed with PFIC type 1 2)Hospitalised- or out-patients 3)Those who can intake medicine per oral and continue the treatment 4)Those who can receive the observation necessary in this study 5) Those who have given written informed consent on the use of their data for the study (patients or legally acceptable representative)

Exclusion criteria

Exclusion criteria: 1)Those who participated in another study within 3 months 2)Those who have hypersensitivity with phenylbutylate and/or its metabolite

Design outcomes

Primary

Measure	Time frame
To evaluate itching before and after 12-months treatment with sodium phenylbutylate	—

Secondary

Measure	Time frame
1) Physical findings 2) Biomarkers such as AST, ALT, gGTP, T.Bil, D.Bil 3) Biomarkers such as plasma amino acid fraction (isoleucine, glutamine, glutamic acid, argineine), and anmonia 4) Dermatology Life Quality Index 5) Pharmacokinetics 6) Adverse events	—

Countries

Japan

Contacts

Public ContactHiroki Kondou

Kindai University Nara Hospital Department of Pediatrics

kondou-hiroki@med.kindai.ac.jp0743-77-0880

Outcome results

None listed

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