Home › Trials › NCT05035030

Long-term safety and effectiveness of odevixibat for Alagille syndrome

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

Phase 3 Interventional Ipsen · NCT05035030

This study is testing if a new medication called odevixibat is safe and effective for people with Alagille syndrome over a long period of time.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment70 (estimated)
SexAll
SponsorIpsen Industry-sponsored
Locations39 sites (San Diego, California and 38 other locations)
Trial IDNCT05035030 on ClinicalTrials.gov

What this trial studies

This Phase 3, open-label, multi-center study evaluates the long-term safety and efficacy of odevixibat in patients with Alagille syndrome, a rare genetic disorder affecting multiple organ systems. Participants are divided into two cohorts: those who have previously participated in a related study and infants under 12 months of age. The study includes a treatment period of up to 72 weeks, followed by an optional extension for continued treatment, and a safety follow-up period. Participants will undergo regular clinical evaluations and complete e-diaries to monitor their health and treatment effects.

Who should consider this trial

Good fit: Ideal candidates include infants under 12 months of age with clinically confirmed Alagille syndrome or those who have completed a prior related study.

Not a fit: Patients with Alagille syndrome who are older than 12 months and have not participated in the previous study may not benefit from this trial.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for managing symptoms of Alagille syndrome, particularly cholestatic pruritus.

How similar studies have performed: Other studies have shown promise in treating cholestatic pruritus in similar patient populations, making this approach potentially beneficial.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

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
3. Gestational age ≥36 weeks. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required .
4. Signed parent/legal guardian informed consent.

Exclusion Criteria:

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
   2. Progressive familial intrahepatic cholestasis (PFIC)
   3. Benign recurrent intrahepatic cholestasis
2. 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
3. Patient with past medical history or ongoing chronic diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae
4. Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant chronic infection
5. 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
6. Cancer diagnosis (except for basal cell carcinoma)
7. Chronic kidney disease with an impaired renal function and a glomerular filtration rate \<70 mL/min/1.73 m2
8. Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to the Screening Visit
9. Patient has had a liver transplant, or a liver transplant is planned within 6 months of Study Day 1
10. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
11. 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)
12. Serum alanine aminotransferase (ALT) \>10 × upper limit of normal (ULN) at Screening
13. Serum ALT \>15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
14. Total bilirubin \>15 × ULN at Screening
15. 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.
16. Patient exposed to alcohol or substance abuse in utero
17. Bile acid or lipid binding resins and medications that slow gastrointestinal motility
18. 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
19. 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

Where this trial is running

San Diego, California and 38 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Alagille Syndrome
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.