Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

Study Overview
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Brief Summary
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An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

Detailed Description
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Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Official Title
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An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT-EXT)

Conditions
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Alagille Syndrome

Intervention / Treatment
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* Drug: Odevixibat


Participation Criteria
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Eligibility Criteria
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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 hormonal contraception, 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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Odevixibat (A4250)<br>Capsules for oral administration once daily for 72 weeks. | Drug: Odevixibat<br>* Odevixibat is a small molecule and selective inhibitor of IBAT.<br>* Other names: A4250;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in pruritus | Change from baseline in scratching score as measured by the Albireo Observer-Reported Outcome Caregiver Instrument | Baseline to week 72 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in serum bile acids | Change from baseline in serum bile acids | Baseline to week 72 | 
| Change in Quality of Life | Change from baseline in quality-of-life assessment as measured by the Pediatric Quality of Life Inventory Questionnaire | Baseline to week 72 | 
| Change in Sleep Parameters | Change from baseline in sleep parameters as measured by the Albireo Patient and Observer-Reported Outcome instruments | Baseline to week 72 | 
| Change in Global Symptom Relief | Change from baseline in global symptom relief as measured by both the Global Impression of Symptoms and Global Impression of Change questionnaires | Baseline to Weeks, 4, 12, 24, 48, and 72 | 
| Safety and Tolerability | Safety and Tolerability as assessed by the Number of Participants with Treatment-emergent Adverse Events as Assessed by CTCAE v5.0 | Baseline to week 72 |