Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

March 6, 2024 updated by: Albireo

Odevixibat (A4250) for the Treatment of Progressive Familial Intrahepatic Cholestasis (Expanded Access Program)

To provide treatment access to patients with PFIC in the US who have pruritus and elevated serum bile acids and who are not able to enroll in A4250-008 (PEDFIC2) for the following reasons: 1) Do not meet eligibility criteria for PEDFIC 2; 2) Are not able to get to a PEDFIC 2 site for geographical reasons, and 3) Do meet the eligibility criteria for PEDFIC 2 after recruitment has been completed

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Detailed Description

Eligible patients will be enrolled into this expanded-access program and treated with an oral dose of 120 μg/kg/day of odevixibat and evaluated on an ongoing basis.

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02109
        • Albireo Pharma Inc.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Albireo

Investigators

  • Study Director: Ipsen Medical Director, Ipsen

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

July 13, 2020

First Submitted That Met QC Criteria

July 20, 2020

First Posted (Actual)

July 23, 2020

Study Record Updates

Last Update Posted (Actual)

March 7, 2024

Last Update Submitted That Met QC Criteria

March 6, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • A4250-014

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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