A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome

Phase 1

Recruiting

Conditions: Alagille Syndrome
MedDRA version: 20.0Level: PTClassification code: 10053870Term: Alagille syndrome Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: CTIS2023-509028-17-00

Lead Sponsor: Albireo AB

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 50

Inclusion Criteria

Completion of the 24-week Treatment Period of Study A4250-012;, Signed 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 to remain on the study;, Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study;, Sexually active males and females must agree to use a reliable contraceptive method with = 1% failure rate (such as intra-uterine device or complete abstinence) from signed informed consent through 90 days after last dose of study drug;

Exclusion Criteria

Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy;, Patients who were not compliant with study drug treatment or procedures in Study A4250-012;, Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study;, Known hypersensitivity to any components of odevixibat;

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT);Secondary Objective: To demonstrate a sustained effect of odevixibat on serum bile acids in patients with ALGS who have completed study A4250-012, To evaluate an effect of odevixibat on parameters related to quality of life, To evaluate the long-term safety and tolerability of repeated daily doses of odevixibat in patients with ALGS;Primary end point(s): Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.

Secondary Outcome Measures