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

APPROVED_FOR_MARKETING

Conditions studied: Alagille Syndrome

In brief

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.

Key facts

Study ID

NCT04530994

Run by

Mirum Pharmaceuticals, Inc.

Last updated by the study team

2021-10-20

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of California, Los Angeles — Los Angeles, California, United States
• Stanford University — Redwood City, California, United States
• University of California San Francisco — San Francisco, California, United States
• Georgetown University — Washington D.C., District of Columbia, United States
• Advent Health — Orlando, Florida, United States
• Children's Healthcare of Atlanta/Emory University — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital — Chicago, Illinois, United States
• Ochsner Clinic Foundation — New Orleans, Louisiana, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• The Children's Hospital at Montefiore — New York, New York, United States
• University of Oklahoma — Oklahoma City, Oklahoma, United States
• Baylor College of Medicine — Houston, Texas, United States
• University of Virginia — Charlottesville, Virginia, United States
• Medical College of Wisconsin — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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