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

Phase 1

Conditions: Alagille Syndrome
MedDRA version: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2021-000996-36-FR

Lead Sponsor: Albireo AB

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 60

Inclusion Criteria

1. Completion of the 24-week Treatment Period of Study A4250-012;
2. 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;
3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study;
4. Sexually active males and females must agree to use a reliable contraceptive method with = 1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) from signed informed consent through 90 days after last dose of study drug;
Are the trial subjects under 18? yes
Number of subjects for this age range: 45
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy;
2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012;
3. 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;
4. 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;<br>To evaluate an effect of odevixibat on parameters related to quality of life;<br>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.;Timepoint(s) of evaluation of this end point: Change from baseline in scratching through Week 72

Secondary Outcome Measures

Name	Time	Method

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