Currently enrolling clinical trials for ALGS
Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (ASSERT)
This double-blind, placebo-controlled Phase 3 is investigating the efficacy and safety of odevixibat compared to placebo in 63 patients with Alagille Syndrome (ALGS). Participants will receive either odevixibat or placebo daily for 24 weeks.
Study inclusion criteria include a genetically confirmed diagnosis of ALGS, history of pruritus, and elevated serum bile acid levels. Exclusion criteria include history of liver transplant/liver transplant planned within 6 months of randomization, history of other types of liver disease, ALT >10× upper limit of normal (ULN) at screening, uncontrolled/recalcitrant pruritic condition other than ALGS, total bilirubin >15 × ULN at screening.
The estimated study completion date is July 2022.
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
In this Expanded Access study, patients 12 months and older with ALGS with cholestatic pruritus will be treated with 400 µg/kg/day of maralixibat. Safety and tolerability will be evaluated.
Inclusion criteria include, but are not limited to, clinical diagnosis of ALGS and No other therapeutic options or access to any ongoing ALGS clinical trials. Exclusion criteria include pregnant or breastfeeding women; a medical history of compensated or decompensated cirrhosis; or presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action.
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)
In this open-label, interventional Phase 2 study, approximately 12 participants with cholestatic liver disease (ALGS or PFIV) will receive maralixibat up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks to evaluate the safety and efficacy of this treatment in infants.
Inclusion criteria include bodyweight of ≥2.5 kg; ≥31 days and <12 months of age at the baseline visit; gestational age ≥36 weeks at birth; and diagnosis of PFIC or ALGS. Exclusion criteria include a history of surgical disruption of the enterohepatic circulation; history of liver transplant or imminent need for liver transplant; decompensated cirrhosis presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs; or presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant’s participation in or completion of the study.
The study is estimated to be completed in January 2023.
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC) (LOGIC)
In this observational study, 1675 participants, include 400 with ALGS, will be followed for at least 20 years, consisting of a baseline visit and 20 annual follow-up visits, to investigate the natural history and progression of 4 cholestatic liver diseases, including ALGS.
Inclusion criteria include siblings of participants with alpha-1-antitrypsin deficiency, who themselves have alpha-1-antitrypsin deficiency of liver disease; meets the enrollment criteria for 1 of the 4 cholestatic liver diseases; and Children and young adults diagnosed with one of the four cholestatic diseases from birth through 25 years old. Exclusion criteria include inability to comply with the longitudinal follow-up and failure to provide an informed consent document.
The estimated study completion date is May 2024.