Clinical trial NCT04530994: A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
Official title: A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome
Study type: EXPANDED_ACCESS
Overall status: APPROVED_FOR_MARKETING
Conditions: Alagille Syndrome
Keywords: Liver Disease
ALGS
Maralixibat
Bile Duct Diseases
Digestive System Diseases
Biliary Tract Diseases
Cholestasis
Pruritis
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Clinigen, Inc.
First posted: 2020-08-28
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California, Los Angeles
state: California
city: Redwood City
country: United States
facility: Stanford University
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: Orlando
country: United States
facility: Advent Health
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta/Emory University
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital
state: Illinois
city: New Orleans
country: United States
facility: Ochsner Clinic Foundation
state: Louisiana
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: New York
country: United States
facility: The Children's Hospital at Montefiore
state: New York
city: Oklahoma City
country: United States
facility: University of Oklahoma
state: Oklahoma
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
References: