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Long-term Safety and Efficacy of Odevixibat for Alagille Syndrome

This study is looking at the long-term safety and how well odevixibat works for people with Alagille syndrome (ALGS). ALGS is a rare genetic condition that can affect many parts of the body, including the liver, and often causes severe itching (pruritus). Odevixibat is a drug that has already been approved for treating itching in infants with ALGS over 12 months old. This study will include participants who were in a previous study and also infants under 12 months of age. Researchers will be checking for side effects and how much the itching improves over time. The study is currently unclear on its recruitment status and plans to enroll 62 participants.

Study design
This is an open-label study, meaning you and your doctor will know which treatment you are receiving. It includes two groups of participants, totaling 62 people.
What's involved
You would visit the clinic every 4 to 12 weeks during the treatment period, which lasts 72 weeks for some and 12 weeks for others. You would take odevixibat daily and complete an e-diary, questionnaires, blood tests, urine collections, and physical exams.
Compensation
Not stated in the trial record.
Follow-up
After the treatment period, there is a safety follow-up period of 4 weeks for some participants and 2 weeks for others, unless you continue into an optional extension period.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05035030

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

Active, Not Recruiting
PHASE3All AgesInterventionalTreatment
Albireo, an Ipsen Company
~62 participants
Updated 2026-08-31 on ClinicalTrials.gov
What's tested:Odevixibat

At a glance

Recruiting sites
0 of 35 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change from baseline in pruritus
Measured over Baseline to week 72 (cohort 1).
+6 more outcomes measured

Conditions

Alagille Syndrome

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Alagille Syndrome

NCT05035030

Where you'd take part

This study runs at 35 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Antenne pediatrique du CIC-Hopital Jeanne De Flandre

    Lille, Franceno site contact published

  • AOU Meyer

    Florence, Italyno site contact published

  • Atrium Health Carolinas Medical

    Durham, North Carolinano site contact published

  • Azienda Ospedale University

    Padova, Italyno site contact published

  • Birmingham Women's and Children's NHS Foundation Trust

    Birmingham, United Kingdomno site contact published

  • Boston Children's Hospital

    Boston, Massachusettsno site contact published

  • Charité - Universitätsmedizin Berlin

    Berlin, Germanyno site contact published

  • Children's Healthcare of Atlanta

    Atlanta, Georgiano site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Ipsen Medical Director · STUDY_DIRECTOR · Ipsen

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

What this trial measures

  • Change from baseline in pruritusBaseline to week 72 (cohort 1).

    Assessed as change in scratching score as measured by measured by the Albireo Observer-Reported Outcome Caregiver Instrument.

  • Percentage of participants with Treatment Emergent Adverse Event (TEAEs) and Serious Adverse Events (SAEs)Baseline to week 12 (cohort 2).

    An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. A serious adverse event (SAE) is an AE that results in any of following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. Treatment-emergent adverse events. TEAEs included both Serious TEAEs and non-serious TEAEs.

  • Percentage of participants with clinically significant changes from baseline in Physical ExaminationBaseline to week 12 (cohort 2).

    The clinical significance will be graded by the investigator.

  • Percentage of participants with clinically significant changes in Laboratory ParametersBaseline to week 12 (cohort 2).

    The following laboratory parameters will be reported: blood chemistry, hematology and coagulation. The clinical significance will be graded by the investigator.

  • Percentage of participants with clinically significant changes from baseline in Vital Signs.Baseline to week 12 (cohort 2).

    The clinical significance will be graded by the investigator.

  • Change from baseline in concomitant medications.Baseline to week 12 (cohort 2).
  • Change from baseline in fat-soluble vitamin levels.Baseline to week 12 (cohort 2).