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Promising long-term results for rare liver disease drug in kids

NCT ID NCT03659916

Disease control Sponsor: Albireo, an Ipsen Company Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 2 times

Summary

This study looked at the long-term safety and effectiveness of a drug called A4250 (odevixibat) in 116 children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease. The children took the drug for up to 72 weeks. The study measured changes in bile acid levels and itching. Results suggest the drug may help control the disease over time, but it is not a cure.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

116 people

The number who actually took part.

Started

Sep 2018

Finished

Dec 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 months to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria Cohort 1: 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. Patients who withdrew from Study A4250-005 due to a study drug related adverse event were not eligible. 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 Inclusion Criteria Cohort 2: 1. A male or female patient of any age, with a clinical diagnosis of PFIC, including episodic forms (i.e., benign recurrent intrahepatic cholestasis \[BRIC\]), 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. 7. Age appropriate patients are expected to have a consistent caregiver for the duration of the study 8. Caregivers and age-appropriate patients (≥8 years of age) must be willing and able to use an eDiary device as required by the study Exclusion Criteria Cohort 1: 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 Exclusion Criteria Cohort 2: 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. 4. Patient with past medical history or ongoing chronic (i.e., \>3 months) diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae. 5. Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant, acute, or chronic infection, or past medical history of any major episode of infection requiring hospitalization or treatment with parenteral anti-infective treatment within 4 weeks of treatment start (Study Day 1) or completion of oral anti-infective treatment within 2 weeks prior to start of Screening Period. 6. Any patient with suspected or confirmed cancers except for basal cell carcinoma, and non-liver cancers treated at least 5 years prior to Screening with no evidence of recurrence. 7. Patient has had a liver transplant, or a liver transplant is planned within 6 months of the Screening/Inclusion Visit. 8. Decompensated liver disease, coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy 9. International normalized ratio (INR) \>1.4 (the patient may be treated with Vitamin K intravenously, and if INR is ≤1.4 at resampling the patient may be included). 10. Serum alanine aminotransferase (ALT) \>10 × upper limit of normal (ULN) at Screening. 11. Serum ALT \>15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation. 12. Total bilirubin \>10 × ULN at Screening. 13. Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC. Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases. 14. Any patient who is pregnant or lactating or who is planning to become pregnant within 72 weeks of the Screening/Inclusion Visit. 15. Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intrauterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter (from signed informed consent through 90 days after last dose of study drug). 16. Patient with a past medical history of alcohol or substance abuse will be excluded. Patient must agree to refrain from illicit drug and alcohol use during the study. 17. Administration of bile acid or lipid binding resins and medications that slow gastrointestinal (GI) motility. 18. Patient has had investigational exposure to a drug, biologic agent, or medical device within 30 days prior to Screening, or 5 half-lives of the study agent, whichever is longer. 19. 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.

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Conditions

The condition(s) this trial relates to.

benign recurrent intrahepatic cholestasis cholestasis Cholestasis, progressive familial intrahepatic 1 progressive familial intrahepatic cholestasis progressive familial intrahepatic cholestasis type 1 progressive familial intrahepatic cholestasis type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Progressive familial intrahepatic cholestasis

Contacts and locations

Locations

  • Akdeniz University

    Antalya, Turkey (Türkiye)

  • Astrid Lindgren Children's Hospital, Karolinska University Hospital

    Solna, Sweden

  • Azienda Ospedaliera Papa Giovanni XXIII

    Bergamo, Italy

  • Baylor College of Medicine - Texas Children's Liver Center

    Houston, Texas, 77030, United States

  • Birmingham Women's and Children's NHS Foundation Trust

    Birmingham, United Kingdom

  • Children's Hospital Colorado

    Denver, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Cliniques Universitaires Saint-Luc

    Woluwe-Saint-Lambert, Belgium

  • Columbia University Medical Center - Presbyterian Hospital Building

    New York, New York, 10032, United States

  • Emory University School of Medicine

    Atlanta, Georgia, 30329, United States

  • Gazi University

    Ankara, Turkey (Türkiye)

  • Hacettepe University Faculty of Medicine

    Ankara, Turkey (Türkiye)

  • Hospital De La Timone

    Marseille, France

  • Hospital Necker-Enfants Maladies

    Paris, France

  • Hospital Universitari Vall d'Hebron

    Barcelona, Spain

  • Hospital Universitario La Paz

    Madrid, Spain

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Inonu University Medical Faculty

    Malatya, Turkey (Türkiye)

  • Institute of Liver Studies - Kings College Hospital

    London, United Kingdom

  • Instytut Pomnik - Centrum Zarowia Dziecka

    Warsaw, Poland

  • Istanbul University Medical Faculty

    Istanbul, Turkey (Türkiye)

  • Johns Hopkins School of Medicine

    Baltimore, Maryland, 21287, United States

  • Kinderklinik Tubingen, Universitatsklinikum Tubingen

    Tübingen, Germany

  • King Faisal Specialist Hospital & Research Centre

    Riyadh, Saudi Arabia

  • Leeds General Infirmary

    Leeds, United Kingdom

  • Medizinische Hochschule Hannover

    Hanover, Germany

  • Ospedale Regina Margherita

    Torino, Italy

  • Riley Hospital for Children - Riley Children's Specialists

    Indianapolis, Indiana, 46202, United States

  • Schneider Children's Medical Center Of Israel

    Petah Tikva, Israel

  • Shaare-Zedek Mc

    Jerusalem, Israel

  • The Hospital for Sick Children

    Toronto, Canada

  • The Royal Children's Hospital

    Melbourne, Australia

  • Universitair Medisch Centrum (UMC) Utrecht

    Utrecht, Netherlands

  • Universite Paris SUD - Hpitaux Universitaires Paris-Sud - Hopital Bicetre

    Le Kremlin-Bicêtre, France

  • University Hospital Of Padova

    Padova, Italy

  • University Medical Center Groningen

    Groningen, Netherlands

  • University and Pediatric Hospital of Lyon

    Bron, France

  • Univesitatsklinikum Tubingen Klinik fur Kinder und Jugendmedizin

    Tübingen, Germany

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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