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

Led by Albireo, an Ipsen Company · Updated on 2026-07-02

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

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.

CONDITIONS

Brief Title

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

Who Can Participate

All Genders

Eligibility Criteria

Inclusion Criteria:

Cohort 1 :

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.

Cohort 2 :

Infant with clinically confirmed ALGS , ≤11 months of age at Study Day 1
Body weight ≥2 kg at Study Day 1
Gestational age ≥36 weeks. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required .
Signed parent/legal guardian informed consent.

Exclusion Criteria:

Cohort 1 :

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

Cohort 2 :

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
2. Progressive familial intrahepatic cholestasis (PFIC)
3. Benign recurrent intrahepatic cholestasis
Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to, inflammatory bowel disease
Patient with past medical history or ongoing chronic diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae
Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant chronic infection
Recent infection requiring hospitalization or treatment with parenteral anti-infective within 4 weeks of Study Day 1 or completion of oral anti-infective treatment within 2 weeks prior to the Screening Visit
Cancer diagnosis (except for basal cell carcinoma)
Chronic kidney disease with an impaired renal function and a glomerular filtration rate <70 mL/min/1.73 m2
Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to the Screening Visit
Patient has had a liver transplant, or a liver transplant is planned within 6 months of Study Day 1
Decompensated liver disease, 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, and if INR is ≤1.4 at resampling the patient may be enrolled)
Serum alanine aminotransferase (ALT) >10 × upper limit of normal (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 >15 × ULN at Screening
Patient suffers from uncontrolled, recalcitrant pruritic condition other than ALGS. Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases.
Patient exposed to alcohol or substance abuse in utero
Bile acid or lipid binding resins and medications that slow gastrointestinal motility
Patient has had investigational exposure to a drug, biologic agent, or medical device within 30 days prior to the Screening Visit, or 5 half-lives of the study agent, whichever is longer
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

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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

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria