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Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC | Clinical Trials | Clareo Health

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Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)

NCT03659916•Albireo, an Ipsen Company

View on ClinicalTrials.gov

Summary

Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in children with PFIC.

Eligibility

Age

0 - 100 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of intolerable symptoms after completing at least 12 weeks of treatment
•2. Signed informed consent and assent as appropriate
•3. Patients expected to have a consistent caregiver for the duration of the study
•4. Caregivers (and age appropriate patients) must be willing and able to use an eDiary device as required by the study
•1. A male or female patient of any age, with a clinical diagnosis of PFIC, including episodic forms (i.e., BRIC), and with a body weight ≥5 kg at Visit S-1.
•2. Patient must have clinical genetic confirmation of PFIC
•3. Patients with PFIC, excluding BRIC, must have elevated serum bile acid concentration,specifically measured to be ≥100 μmol/L, taken as the average of 2 samples at least 7 days apart (Visits S-1 and S-2) prior to the Screening/Inclusion Visit (Visit 1).
•4. Patients with PFIC, excluding BRIC, must have history of significant pruritus and a caregiver-reported observed scratching or patient-reported itching (for patients \>18 with no caregiver-reported observed scratching) in the eDiary average of ≥2 (on 0 to 4 scale) in the 2 weeks prior to the Screening/Inclusion Visit (Visit 1).
•5. Patients with episodic forms of PFIC (i.e., BRIC) must have an emerging flare characterized by clinically significant pruritus and elevated serum bile acid levels/cholestasis as judged by the investigator.
•6. Patient and/or legal guardian must sign informed consent (and assent) as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent in order to remain in the study.
+2 more criteria

Exclusion Criteria

•1. Decompensated liver disease: coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
•2. Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter
•3. Patients not compliant with treatment in study A4250-005
•4. Any other conditions or abnormalities which, in the opinion of the investigator or Medical Monitor, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
•1. Known pathologic variations of the ABCB11 gene that have been demonstrated to result in complete absence of the BSEP protein
•2. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
•1. Biliary atresia of any kind
•2. Suspected or proven liver cancer or metastasis to the liver on imaging studies
•3. Histopathology on liver biopsy is suggestive of alternate non-PFIC related etiology of cholestasis Note: Patients with clinically significant portal hypertension are allowed.
•3. Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to,inflammatory bowel disease.
+17 more criteria

Locations (41)

Children's Hospital Los Angeles

Los Angeles, California, United States

Children's Hospital Colorado

Denver, Colorado, United States

Emory University School of Medicine

Atlanta, Georgia, United States

Riley Hospital for Children - Riley Children's Specialists

Indianapolis, Indiana, United States

Johns Hopkins School of Medicine

Baltimore, Maryland, United States

Washington University School of Medicine

St Louis, Missouri, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Columbia University Medical Center - Presbyterian Hospital Building

New York, New York, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Key Dates

Start Date

September 28, 2018

Primary Completion Date

February 15, 2024

Completion Date

December 2, 2025

Last Updated

March 2, 2026

Enrollment

116

ACTUAL participants

Conditions

Progressive Familial Intrahepatic Cholestasis

Interventions

A4250 (odevixibat)

DRUG

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea

NCT07185919

Progressive Familial Intrahepatic Cholestasis

View study

RECRUITING

A Study to Assess the Genetic Variations in Bile Flow Disorders: Linking Progressive Familial Intrahepatic Cholestasis (PFIC)-Related Genes to Symptoms in Adults With Recurrent Cholestasis in Spain

NCT07191704

PFIC - Progressive Familial Intrahepatic Cholestasis

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 2, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC

Inclusion Criteria

Exclusion Criteria

A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea

A Study to Assess the Genetic Variations in Bile Flow Disorders: Linking Progressive Familial Intrahepatic Cholestasis (PFIC)-Related Genes to Symptoms in Adults With Recurrent Cholestasis in Spain

A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)

Prospective Analysis of the Treatment of Progressive Familial Intrahepatic Cholestasis (TreatFIC)

NAtural Course and Prognosis of PFIC and Effect of Biliary Diversion

A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384