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Bile Acid Sequestrant

A4250 high dose for Progressive Familial Intrahepatic Cholestasis (PEDFIC 1 Trial)

Phase 3
Waitlist Available
Research Sponsored by Albireo
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up over 24 weeks of treatment
Awards & highlights
All Individual Drugs Already Approved
Pivotal Trial

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.

Eligible Conditions
  • Progressive Familial Intrahepatic Cholestasis
  • Progressive Familial Intrahepatic Cholestasis 1

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~over 24 weeks of treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and over 24 weeks of treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Percentage of Participants Experiencing at Least a 70% Reduction in Fasting s-BA Concentration From Baseline to the End of Treatment or Reaching a Level <= 70 μmol/L After 24 Weeks of Treatment (European Union and Rest of the World Primary Endpoint)
Proportion of Positive Pruritus Assessments at the Participant Level Over the 24-week Treatment Period Based on the Albireo Observer-reported Outcome (ObsRO) Instrument (United States Primary Endpoint)

Side effects data

From 2020 Phase 3 trial • 62 Patients • NCT03566238
26%
Pyrexia
26%
Upper respiratory tract infection
21%
Diarrhoea
16%
Alanine aminotransferase increased
16%
Vomiting
11%
Nasopharyngitis
11%
Otitis media
11%
Vitamin D deficiency
11%
Cough
11%
Splenomegaly
11%
Blood bilirubin increased
11%
Blood alkaline phosphatase increased
5%
Vitamin D decreased
5%
Otorrhoea
5%
Liver function test increased
5%
Respiratory tract infection
5%
Cardiac ablation
5%
Cholelithiasis
5%
Urinary tract infection
5%
Supraventricular tachycardia
5%
Abdominal pain
5%
Mouth ulceration
5%
Viral infection
5%
Vitamin A deficiency
5%
Vitamin E deficiency
5%
Cystitis haemorrhagic
5%
Rhinitis allergic
5%
Dehydration
5%
Ear pain
5%
Eye discharge
5%
Abdominal discomfort
5%
Gastroenteritis norovirus
5%
Influenza
5%
Aspartate aminotransferase increased
5%
Platelet count increased
5%
Blood creatine phosphokinase increased
5%
Irritability
5%
Genital rash
5%
Epistaxis
5%
Dermatitis allergic
5%
Pruritus
5%
Sinusitis bacterial
5%
Abdominal pain upper
5%
Dental caries
5%
Hepatomegaly
5%
Parotitis
5%
Headache
5%
Dizziness
100%
80%
60%
40%
20%
0%
Study treatment Arm
A4250 High Dose
A4250 Low Dose
Placebo

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: A4250 low doseExperimental Treatment1 Intervention
Capsules for oral administration (40 ug/kg) once daily for 24 weeks
Group II: A4250 high doseExperimental Treatment1 Intervention
Capsules for oral administration (120 ug/kg) once daily for 24 weeks
Group III: PlaceboPlacebo Group1 Intervention
Capsules for oral administration (to match active) once daily for 24 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Odevixibat
FDA approved

Find a Location

Who is running the clinical trial?

AlbireoLead Sponsor
16 Previous Clinical Trials
1,138 Total Patients Enrolled
~8 spots leftby Dec 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
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