Clinical Trials in the USA / NCT06850038
Recruiting
Observational
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
NCT06850038 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Alagille Syndrome
Study summary
This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.
Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching.
The main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.
Eligibility
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
Primary outcome measure(s)
- Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation. — From first dose to end of study (approximately 5 years data collection)
- Surgical biliary diversion-free survival — From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death. - Liver transplant-free survival — From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death. - Overall survival — From first dose to end of study (approximately 5 years data collection)
Defined as time from the start of odevixibat treatment to death.
Trial sites (10)
| Facility | City | Region | Status |
|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | Recruiting |
| UCSF Benioff Children's Hospital | San Francisco | California | Recruiting |
| The Children's Mercy Hospital | Kansas City | Missouri | Recruiting |
| SSM Health Cardinal Glennon Children's Hospital | St Louis | Missouri | Recruiting |
| NYU Langone - NYU Grossman School of Medicine | New York | New York | Recruiting |
| Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presbyterian (MS-CHONY) | New York | New York | Recruiting |
| Le Bonheur Children's Hospital | Memphis | Tennessee | Recruiting |
| Children's Medical Center Of Dallas | Dallas | Texas | Recruiting |
| Proactive El Paso,LLC | El Paso | Texas | Recruiting |
| Primary Children's Hospital (PCH) - Intermountain Healthcare/University of Utah | Salt Lake City | Utah | Not Yet Recruiting |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06850038 on ClinicalTrials.gov ↗ ← All trials in the USA