Alagille Syndrome Clinical Trials

7 recruitingLast updated: May 13, 2026

There are 7 actively recruiting alagille syndrome clinical trials across 16 countries. Studies span Phase 3, Phase 4. Top locations include Kansas City, Missouri, United States, Groningen, Netherlands, Atlanta, Georgia, United States. Updated daily from ClinicalTrials.gov.


Alagille Syndrome Trials at a Glance

7 actively recruiting trials for alagille syndrome are listed on ClinicalTrialsFinder across 6 cities in 16 countries. The largest study group is Phase 3 with 1 trial, with the heaviest enrollment activity in Kansas City, Groningen, and Atlanta. Lead sponsors running alagille syndrome studies include Mirum Pharmaceuticals, Inc., Albireo, an Ipsen Company, and Children's Hospital of Eastern Ontario.

Browse alagille syndrome trials by phase

About Alagille Syndrome Clinical Trials

Looking for clinical trials for Alagille Syndrome? There are currently 5 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Alagille Syndrome trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Alagille Syndrome clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 17 of 7 trials

Recruiting
Phase 3

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

Alagille Syndrome
Albireo, an Ipsen Company70 enrolled39 locationsNCT05035030
Recruiting

A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

Alagille Syndrome
Ipsen30 enrolled9 locationsNCT06850038
Recruiting

Pediatric Evaluation and Registry for Liver Cholestasis in Canada

PFIC - Progressive Familial Intrahepatic CholestasisAlagille Syndrome (ALGS)Cholestasis, Intrahepatic
Children's Hospital of Eastern Ontario220 enrolled13 locationsNCT07411716
Recruiting

A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)

Progressive Familial Intrahepatic Cholestasis (PFIC)Alagille Syndrome (ALGS)
Takeda50 enrolled1 locationNCT07293897
Recruiting
Phase 4

Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)

Alagille Syndrome
Mirum Pharmaceuticals, Inc.100 enrolled13 locationsNCT07290257
Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Multiple Endocrine Neoplasia Type 1Multiple Endocrine Neoplasia Type 2Rare Disorders+337 more
Sanford Health20,000 enrolled2 locationsNCT01793168
Recruiting

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

Progressive Familial Intrahepatic CholestasisAlagille Syndrome
Mirum Pharmaceuticals, Inc.70 enrolled8 locationsNCT06193928