Alagille Syndrome (ALGS)

There is a wide range of Alagille syndrome clinical trials currently underway in the United States and Europe testing approved and investigational therapies for treating the disease, as well as uncovering new insights into the disease’s pathogenesis and improving diagnosis. The following trials highlight some of the most high-profile and late-stage clinical trials for Alagille syndrome therapies. A complete, up-to-date list of all Alagille syndrome clinical trials can be found at

Odevixibat – ASSERT and ASSERT-EXT Trials

Odevixibat is sold under the brand name Bylvay™. It is a small molecule inhibitor of the ileal bile acid transporter (IBAT) used to treat cholestatic diseases. Odevixibat gained US Food and Drug Administration (FDA) approval in 2021 for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged 3 months and older.1  

The safety and efficacy of odevixibat for patients with Alagille syndrome is being investigated in a phase 3 double-blind, randomized, placebo-controlled trial of 63 participants, including children and adults. Patients will receive either oral odevixibat or a placebo for 24 weeks. The primary outcome of the study is the change from baseline in scratching score to month 6 as measured by the Albireo Observer-Reported Outcome scratching score. Secondary endpoints include the change in serum bile acid (sBA) levels from baseline to week 24 and the number of participants with treatment-emergent adverse events and serious adverse events. The expected completion date is July 2022.2 

Recent articles about Odevixibat:

The ASSERT-EXT trial is an open-label study of 63 participants that is enrolling via invitation. It seeks to establish the long-term safety and efficacy of odevixibat in patients with Alagille syndrome. The study duration is prolonged compared to the ASSERT trial and lasts 72 weeks. The primary outcome measure is change from baseline in scratching score as measured by the Albireo Observer-Reported Outcome Caregiver Instrument from baseline to week 72. Secondary outcome measures include change in sBA levels, change in quality of life, change in sleep parameters, change in global symptom relief, and safety and tolerability. The expected completion date is October 2023.3

Read more about Odevixibat

Maralixibat – RISE Trial

Maralixibat is sold under the brand name Livmarli™ and is also an IBAT inhibitor. It gained initial FDA approval in September 2021 for the treatment of cholestatic pruritus in patients with Alagille syndrome 1 year of age and older.4 

Recent articles about Maralixibat:

The RISE trial is a phase 2 open-label study in the recruitment phase that aims to assess the safety of maralixibat in children under 12 months of age with PFIC, Alagille syndrome, or cholestatic liver disease. Participants will receive varying dosages of maralixibat over a 13-week study period. The primary outcome is the frequency of treatment-emergent adverse events through week 13, and the secondary outcome measure is change in fasting sBA levels through week 13. It is expected to be completed in January 2023.5 

Read more about Maralixibat

FibroScan™ – FORCE Trial

Patients with Alagille syndrome usually undergo a liver biopsy as part of the initial diagnostic work-up and evaluation; however, subsequent surveillance liver biopsy is rarely performed due risks associated with invasive procedures. Transient elastography using an ultrasound device called FibroScan™ to obtain liver stiffness measurements (LSMs) may fulfill an unmet and critical need in the management of children with chronic liver disease. The FORCE trial is a cross-sectional, observational study of 458 participants that has been ongoing since October 2016 and aims to evaluate the role of noninvasive FibroScan in pediatric and adult patients up to 21 years of age with biliary atresia, Alagille syndrome, alpha-1 antitrypsin deficiency, portal hypertension, liver fibrosis, and cholestasis. The primary outcome measure is the distribution of LSMs between participants with and without portal hypertension. Secondary outcome measures will assess changes in LSM from baseline at year 1 and year 2 for patients with the diseases listed above. The estimated study completion date is December 2023. 6


1. Deeks ED. Odevixibat: first approval. Drugs. 2021;81(15):1781-1786. doi:10.1007/s40265-021-01594-y

2. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT). December 19, 2020. Updated February 21, 2022. Accessed March 9, 2022.

3. Long-term safety and efficacy of odevixibat in patients with Alagille syndrome (ASSERT-EXT). September 5, 2021. Updated September 30, 2021. Accessed March 9, 2022.

4. Shirley M. Maralixibat: first approval. Drugs. 2022;82(1):71-76. doi:10.1007/s40265-021-01649-0

5. A study to evaluate the safety and tolerability of maralixibat in infant participants with cholestatic liver diseases including progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS). (RISE). January 28, 2021. Updated December 21, 2021. Accessed March 9, 2022. 

6. FibroScan™ in pediatric cholestatic liver disease (FORCE) (FORCE). October 4, 2016. Updated December 14, 2021. Accessed March 9, 2022.

Reviewed by Debjyoti Talukdar, MD, on 3/9/2022.