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BSEP Function Rescue During Childhood Inhereditary Cholestatic Diseases

NCT04531878 · Status: WITHDRAWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL

Last updated 2023-02-09

No results posted yet for this study

Summary

The purpose of the study is to improve the prognosis of inhereditary cholestasis caused by ABCB11 gene mutations by using BSEP function rescue drugs

Conditions

  • Hereditary Diseases
  • Cholestasis, Intrahepatic

Interventions

DRUG

4-Phenylbutyrate

4-phenylbutyrate therapy will be started at a daily dose of 200 mg kg/d divided in 2 oral doses of sodium phenylbutyrate (AMMONAPS, Swedish Orphan Inter AB). In order to get the best effect, the dose will be increased up to a maximum of 500 mg kg/d.

Sponsors & Collaborators

  • Children's Hospital of Fudan University

    lead OTHER

Principal Investigators

  • Jian-She Wang, Ph.D · Children's hospital of Fudan Unviersity

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Months
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-08
Primary Completion
2023-02-08
Completion
2023-02-08

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04531878 on ClinicalTrials.gov