Long-Term Safety Study of Maralixibat in Cholestatic Liver Disease
Research type
Research Study
Full title
MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
IRAS ID
274904
Contact name
Alastair Baker
Contact email
Sponsor organisation
Mirum Pharmaceutical Inc.
Eudract number
2019-002755-42
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 11 months, 23 days
Research summary
Progressive familial intrahepatic cholestasis (PFIC) is a rare liver disease that primarily affects children. It is a disease where there is poor bile flow and substances are retained in the liver that would normally be excreted into bile. It tends to be passed down to a child from the parents, and gets worse over time. PFIC is associated with short life expectancy and devastating consequences on patients' quality of life.
Alagille syndrome (ALGS) is an inherited cholestatic liver disease in children. Patients with ALGS have too much bile acid in their liver and body that can cause problems with the liver, and cause itching with or without jaundice.
There is currently no approved treatment for PFIC or ALGS and available treatments have limited efficacy. Maralixibat is an investigational medicine (not yet approved by regulatory authorities) which will be given to participants twice a day as an oral solution. This study is a long-term follow up to previous maralixibat studies, designed to evaluate the long-term safety of maralixibat, and whether it will relieve or reduce symptoms and itching.
Based on the high unmet medical need, the current absence of approved treatments and the complications with surgical treatment, the overall benign safety profile, and positive benefit-risk ratio of maralixibat show support for this long-term study. The study also aims to provide long-term access to maralixibat, particularly to those patients who benefited from the treatment in previous studies.
REC name
London - Hampstead Research Ethics Committee
REC reference
20/LO/0038
Date of REC Opinion
19 Mar 2020
REC opinion
Further Information Favourable Opinion