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NCT04530994 Clinical Trial

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

Alagille Syndrome Clinical Trial

Official title:
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome

Clinical Trial Details — Status: Approved for marketing

Administrative data
NCT number NCT04530994
Other study ID # MRX-EAP
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information
Verified date October 2021
Source Mirum Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Approved for marketing
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers
Gender All
Age group 12 Months and older
Eligibility 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

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• maralixibat
Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor

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

Sponsors (2)
Lead Sponsor Collaborator
Mirum Pharmaceuticals, Inc. Clinigen, Inc.

Country where clinical trial is conducted

United States, 

See also
  Status Clinical Trial Phase
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Recruiting NCT06193928 - Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)
Completed NCT02160782 - Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS) Phase 2
Completed NCT00007033 - Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease N/A
Suspended NCT00571272 - Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
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Completed NCT02117713 - An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome Phase 2
Completed NCT02131623 - Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
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Completed NCT03082937 - An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects Phase 1
Completed NCT02047318 - An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS) Phase 2
Completed NCT01903460 - Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome Phase 2
Completed NCT01515631 - Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record Review
Enrolling by invitation NCT05846854 - Decreasing Hemorrhage Risk in Children With Alagille Syndrome N/A

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