Extension Study to Evaluate Safety and Efficacy of Maralixibat in PFIC
Research type
Research Study
Full title
An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)
IRAS ID
274909
Contact name
Richard Thompson
Contact email
Sponsor organisation
Mirum Pharmaceuticals Inc.
Eudract number
2019-003395-39
Clinicaltrials.gov Identifier
n/a, n/a
Duration of Study in the UK
3 years, 0 months, 0 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. There is no currently approved treatment for PFIC and available medical approaches 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 an extension to the previous MRX-502 study and is designed to further evaluate the long-term safety of maralixibat. If the study medicine works as expected, it may help to reduce patients' PFIC symptoms and itching.
REC name
London - Hampstead Research Ethics Committee
REC reference
20/LO/0100
Date of REC Opinion
30 Mar 2020
REC opinion
Further Information Favourable Opinion