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

Mirum Pharmaceuticals

Status

Conditions: Alagille Syndrome

Treatments: Drug: maralixibat

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT04530994

MRX-EAP

Details and patient eligibility

About

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 [e-mail] or telephone [phone].

Full description

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.

Sex: All

Ages: 12+ months old

Volunteers: No Healthy Volunteers

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