NCT04483531

Brief Summary

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

Trial Health

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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 13, 2020

Completed
10 days until next milestone

First Posted

Study publicly available on registry

July 23, 2020

Completed
Last Updated

March 7, 2024

Status Verified

March 1, 2024

First QC Date

July 13, 2020

Last Update Submit

March 6, 2024

Conditions

Progressive Familial Intrahepatic Cholestasis

Keywords

PFICpediatricCholestasis

Interventions

OdevixibatDRUG

A4250 is a small molecule and selective inhibitor of IBAT

Also known as: A4250

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • A male or female patient of any age, with a clinical diagnosis of PFIC, and with a body weight ≥5 kg at Screening visit
  • Patient must have a clinical diagnosis of PFIC
  • Patient must have clinically confirmed pruritus
  • Patient must have elevated serum bile acid levels, specifically measured to be ≥2 × the upper limit of normal (ULN) prior to start of medication
  • 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

You may not qualify if:

  • Patient is expected to have a liver transplant within 6 months of Screening
  • Decompensated liver disease, coagulopathy, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
  • 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)
  • Serum ALT \>10 × ULN at Screening
  • Serum ALT \>15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
  • Total bilirubin \>10 × ULN at Screening
  • Any patient who is pregnant, lactating, or planning to get pregnant
  • Patients who qualify for enrollment in other Phase 2 or Phase 3 trials intended to support marketing approval in PFIC

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Albireo Pharma Inc.

Boston, Massachusetts, 02109, United States

Location

MeSH Terms

Conditions

Cholestasis, progressive familial intrahepatic 1Cholestasis

Interventions

odevixibat

Condition Hierarchy (Ancestors)

Bile Duct DiseasesBiliary Tract DiseasesDigestive System Diseases

Study Officials

  • Ipsen Medical Director

    Ipsen

    STUDY DIRECTOR

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 13, 2020

First Posted

July 23, 2020

Last Updated

March 7, 2024

Record last verified: 2024-03

Locations

US(1)