Observing the long-term effects of odevixibat in patients with Alagille syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study collects data from patients diagnosed with Alagille syndrome who are currently receiving treatment with odevixibat (Bylvay). It aims to observe the long-term effectiveness and safety of the medication in real-world settings. Participants will provide information about their experiences while using the drug in their daily lives. The study is observational, meaning it will not involve any experimental interventions but will focus on gathering existing treatment data.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Diagnosed with ALGS.
* On (or starting) active odevixibat treatment.
* Signed informed consent and assent, as appropriate. Consent/assent from the participant or legal representative should be obtained, as appropriate, before any study data collection is conducted. Participants who turn 18 years of age (or legal age per country) while participating in the study will be required to provide consent for themselves.

Exclusion Criteria:

* Currently participating in a clinical trial with odevixibat.
* Currently participating in any interventional clinical trial for ALGS.
* Have any contraindication to odevixibat as per the locally approved label.
* Had liver transplant before enrolment

Where it's running

• Children's Hospital Los Angeles
  Los Angeles, California, United States Recruiting
• UCSF Benioff Children's Hospital
  San Francisco, California, United States Recruiting
• The Children's Mercy Hospital
  Kansas City, Missouri, United States Recruiting
• SSM Health Cardinal Glennon Children's Hospital
  St Louis, Missouri, United States Recruiting
• NYU Langone - NYU Grossman School of Medicine
  New York, New York, United States Recruiting
• Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presbyterian (MS-CHONY)
  New York, New York, United States Recruiting

Study contacts

• Study coordinator: Ipsen Recruitment Enquiries
• Email: clinical.trials@ipsen.com
• Phone: See email

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• See if odevixibat stays safe for kids and adults with Alagille syndrome over the long term

  This study tests whether continuing odevixibat is safe for people with Alagille syndrome who already take the drug in real life.

  Recruiting at 3 sites Ipsen · Bron and 13 other locations · NCT07585097

• Long-term safety and outcomes of Livmarli for Alagille syndrome in the EU

  This study will follow people with Alagille syndrome who take Livmarli to see if it is safe, well tolerated over the long term, and continues to reduce cholestatic itching.

  Recruiting at 13 sites Phase 4 Mirum Pharmaceuticals, Inc. · Brussels and 14 other locations · NCT07290257

• Evaluating the long-term safety and outcomes of Livmarli in patients with liver diseases

  This study is testing how safe and effective the medication Livmarli is for people with Alagille syndrome or Progressive familial intrahepatic cholestasis over a five-year period.

  Recruiting at 8 sites Mirum Pharmaceuticals, Inc. · Los Angeles, California and 7 other locations · NCT06193928

• Registry for patients with rare diseases

  This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.

  Recruiting Sanford Health · Sioux Falls, South Dakota · NCT01793168

• Canadian pediatric registry for Alagille syndrome and genetic intrahepatic cholestasis

  This registry collects long-term health and genetic information from children in Canada who have Alagille syndrome or genetic intrahepatic cholestasis to better track outcomes and support research.

  Recruiting at 2 sites Children's Hospital of Eastern Ontario · Calgary, Alberta and 12 other locations · NCT07411716

• Maralixibat (Livmarli) use in Alagille syndrome and PFIC — Japanese database analysis

  This project will look at Japanese patients with Alagille syndrome or PFIC who received maralixibat (Livmarli) to see if taking the drug is linked to liver problems.

  Recruiting Takeda · Tokyo, Tokyo · NCT07293897

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.