A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
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An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US. Access can be requested through Clinigen Healthcare Ltd via email MirumALGS@clinigengroup.com or telephone +1-877-768-4303.
Condition or disease
The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.
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Layout table for eligibility information
Ages Eligible for Study:
12 Months and older (Child, Adult, Older Adult)
Sexes Eligible for Study:
Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC)
Clinical and/or genetic diagnosis of ALGS
Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS
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
No other therapeutic options or access to any ongoing ALGS clinical trials
Diagnosis with a cholestatic liver disease other than ALGS
Female who is pregnant or breastfeeding
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
Past medical history of compensated or decompensated cirrhosis
Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action