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This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2

NCT03566238 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 62

Last updated 2025-11-28

Study results available
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Summary

Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2.

Conditions

  • PFIC1
  • PFIC2

Interventions

DRUG

A4250 (odevixibat)

A4250 is a small molecule and selective inhibitor of ileal bile acid transporter (IBAT).

DRUG

Placebo

Placebo identical in appearance to active drug (A4250).

Sponsors & Collaborators

  • Albireo

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2018-05-16
Primary Completion
2020-07-27
Completion
2020-07-28
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Canada
  • France
  • Germany
  • Israel
  • Italy
  • Netherlands
  • Poland
  • Saudi Arabia
  • Spain
  • Sweden
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

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 NCT03566238 on ClinicalTrials.gov