Actively Recruiting
Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)
Led by Ipsen · Updated on 2026-05-28
30
Participants Needed
10
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to observe the long-term effects and safety of the medicine odevixibat (Bylvay) in patients with Alagille syndrome (ALGS), a rare liver disease that causes itching. The study focuses on patients who are already receiving or starting treatment with odevixibat to better understand its everyday use and impact over time. Participants will continue their usual odevixibat treatment while researchers collect information over approximately five years. The study does not assign treatments but observes patients as they take odevixibat in real life, tracking any need for biliary diversion surgery, liver transplantation, and overall survival. During the study, participants will be monitored for changes in weight and height, adverse events related to vitamin deficiencies, liver toxicity, bleeding, and other side effects. Data collection will include ongoing assessments starting from the first dose through the study duration and safety follow-up up to 180 days after the last dose. This long-term observation will help understand the drug's effectiveness and safety in everyday practice.
CONDITIONS
Brief Title
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with Alagille syndrome (ALGS)
- Currently receiving or starting active treatment with odevixibat (Bylvay)
- Signed informed consent or assent as appropriate before any study data collection
- Participants who turn legal adult age during the study must provide their own consent
You will not qualify if you...
- Currently participating in a clinical trial involving odevixibat
- Currently participating in any other interventional clinical trial for Alagille syndrome
- Have any contraindication to odevixibat as per local approved label
- Had a liver transplant before enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Approximately 5 years
Participants who are receiving ongoing treatment with odevixibat are observed for effectiveness and safety over an extended period.
Trial Site Locations
Total: 10 locations
1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
2
UCSF Benioff Children's Hospital
San Francisco, California, United States, 94158
Actively Recruiting
3
The Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Actively Recruiting
4
SSM Health Cardinal Glennon Children's Hospital
St Louis, Missouri, United States, 63104
Not Yet Recruiting
5
NYU Langone - NYU Grossman School of Medicine
New York, New York, United States, 10016
Actively Recruiting
6
Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presbyterian (MS-CHONY)
New York, New York, United States, 10032
Actively Recruiting
7
Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
8
Children's Medical Center Of Dallas
Dallas, Texas, United States, 75246
Actively Recruiting
9
Proactive El Paso,LLC
El Paso, Texas, United States, 79902
Actively Recruiting
10
Primary Children's Hospital (PCH) - Intermountain Healthcare/University of Utah
Salt Lake City, Utah, United States, 84113
Not Yet Recruiting
Research Team
I
Ipsen Recruitment Enquiries
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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