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Can a bile acid blocker ease severe itching in a rare liver disease?

NCT ID NCT04483531

Disease control Sponsor: Albireo Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

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
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 Results may not be published yet.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) This study
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This status has not been confirmed recently, so it may be out of date.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 Jul 23, 2026 · Last updated Jul 24, 2026 · Updated 1 time

Summary

This expanded access program offers the experimental drug odevixibat to people with progressive familial intrahepatic cholestasis (PFIC) who have severe itching and high bile acid levels but cannot join the main clinical trial. The drug works by blocking a bile acid transporter in the gut, which may lower bile acids and reduce itching. The program aims to provide treatment access for patients of any age who meet specific criteria.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
an experimental drug called odevixibat (A4250) that blocks a bile acid transporter in the gut
What this could lead to
If it works, this could provide a treatment option for people with PFIC who have severe itching and high bile acid levels.
What could go wrong
This is an expanded access program, not a controlled trial, so results may be less clear. The drug may not relieve symptoms or could cause side effects like diarrhea or liver issues.

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.

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

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: 1. A male or female patient of any age, with a clinical diagnosis of PFIC, and with a body weight ≥5 kg at Screening visit 2. Patient must have a clinical diagnosis of PFIC 3. Patient must have clinically confirmed pruritus 4. Patient must have elevated serum bile acid levels, specifically measured to be ≥2 × the upper limit of normal (ULN) prior to start of medication 5. 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 Exclusion Criteria: 1. Patient is expected to have a liver transplant within 6 months of Screening 2. Decompensated liver disease, coagulopathy, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy 3. 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 started on program medication) 4. Serum ALT \>10 × ULN at Screening 5. Serum ALT \>15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation 6. Total bilirubin \>10 × ULN at Screening 7. Any patient who is pregnant, lactating, or planning to get pregnant 8. Patients who qualify for enrollment in other Phase 2 or Phase 3 trials intended to support marketing approval in PFIC

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Conditions

The condition(s) this trial relates to.

cholestasis Cholestasis, progressive familial intrahepatic 1 progressive familial intrahepatic cholestasis

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

  • Albireo Pharma Inc.

    Boston, Massachusetts, 02109, United States

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