A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

This is an expanded access trial for Alagille Syndrome focused on measuring Liver Disease, ALGS, Maralixibat, Bile Duct Diseases, Digestive System Diseases, Biliary Tract Diseases, Cholestasis, Pruritis Read More

Inclusion Criteria:

1. Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
2. Clinical and/or genetic diagnosis of ALGS
3. Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
4. Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program
5. No other therapeutic options or access to any ongoing ALGS clinical trials

Exclusion Criteria:

1. Diagnosis with a cholestatic liver disease other than ALGS
2. Female who is pregnant or breastfeeding
3. Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program
4. Past medical history of compensated or decompensated cirrhosis
5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action