Observing the long-term effects of odevixibat in patients with Alagille syndrome
Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)
This study looks at how well the medication odevixibat works and its safety for people with Alagille syndrome over a long period of time by collecting their real-life experiences.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 30 (estimated) |
| Sex | All |
| Sponsor | Ipsen Industry-sponsored |
| Locations | 9 sites (Los Angeles, California and 8 other locations) |
| Trial ID | NCT06850038 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with Alagille syndrome who are currently on or starting treatment with odevixibat.
Not a fit: Patients who are currently participating in other clinical trials involving odevixibat or have had a liver transplant prior to enrollment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term safety and effectiveness of odevixibat for patients with Alagille syndrome.
How similar studies have performed: While this study is observational, similar studies on the long-term effects of treatments for rare diseases have shown promising results in understanding patient outcomes.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Los Angeles, California and 8 other locations
- Childrens Hospital Los Angeles — Los Angeles, California, United States (Recruiting)
- UCSF Pediatric Gastroenterology — San Francisco, California, United States (Recruiting)
- The Children's Mercy Hospital — Kansas City, Missouri, United States (Recruiting)
- St Louis University. SSM Cardinal Glennon Childrens Medical Center — St Louis, Missouri, United States (Recruiting)
- Columbia University — New York, New York, United States (Recruiting)
- NYU Langone - NYU Grossman School of Medicine — New York, New York, United States (Recruiting)
- Le Bonheur Children's Hospital — Memphis, Tennessee, United States (Recruiting)
- UT Southwestern Childrens Medical Center — Dallas, Texas, United States (Recruiting)
- Proactive El Paso,LLC — El Paso, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Ipsen Recruitment Enquiries
- Email: clinical.trials@ipsen.com
- Phone: See email
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.