A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alag… (NCT04530994) | Clinical Trial Compass
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A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
United States
Plain-language summary
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.
Who can participate
Age range
12 Months
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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
Exclusion criteria
. 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action