RecruitingAlagille Syndrome

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

Eligible age

All ages

Accepts

All genders

Locations

12 states

Healthy volunteers

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About this study

The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS). The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver, heart, skeleton, eyes and kidneys. Common symptoms, which often develop during the first three months of life, include blockage of the flow of bile from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), poor weight gain and growth and severe itching (pruritis). The drug used for the study is odevixibat and was authorized for the treatment of cholestatic pruritus in infants with ALGS over 12 months of age by the United States Food and Drug Administration on 13 June 2023.

Sponsor: Albireo, an Ipsen Company

You may qualify if…

✓ Cohort 1 :
✓ 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.
✓ Cohort 2 :
✓ 1. Infant with clinically confirmed ALGS , ≤11 months of age at Study Day 1
✓ 2. Body weight ≥2 kg at Study Day 1

You may not qualify if…

✕ Cohort 1 :
✕ 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
✕ Cohort 2 :
✕ 1. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
✕ 1. Biliary atresia of any kind