Phase 2 - MBX-8025 in Participants with Primary Biliary Cholangitis
Research type
Research Study
Full title
An 8-week, dose ranging, open label, randomized, Phase 2 study with a 44-week extension, to evaluate the safety and efficacy of MBX-8025 in subjects with Primary Biliary Cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA
IRAS ID
215399
Contact name
Douglas Thornburn
Contact email
Sponsor organisation
CymaBay Therapeutics, Inc.
Eudract number
2016-002996-91
Duration of Study in the UK
1 years, 3 months, 2 days
Research summary
The purpose of this research study is to compare the effectiveness and safety of MBX-8025 on Primary Biliary Cholangitis (PBC) when available treatment with ursodeoxycholic acid (UDCA) has proved inadequate or not tolerated. This will be assessed by looking at how the medication affects PBC. The study will assess how MBX-8025 works on the body by using blood tests and questionnaires.
PBC is a serious and potentially life threatening autoimmune disease of the liver characterised by impaired bile flow and accumulation of toxic bile acids.
The study medication works by targeting and initiating a response from a particular protein in the body which is involved in lipid processing in the liver and muscle. The study medication has shown in laboratory and previous human studies that it might be useful in treating the signs and symptoms of certain lipid disorders. It is believed, that PBC patients may also potentially benefit from this new study medication as PBC is closely linked to lipid metabolism.
This study invites participants between 18 to 75 years of age, which have a diagnosis of PBC and are on a stable dose of ursodesoxycholic acid (UDCA) for the past 12 months but do not respond well to UDCA or have an intolerance to UDCA.
Approximately 36 participants at 30 sites in 4 countries (United States, Canada, United Kingdom and Germany) will participate in this research study. This is an open label study. This means that the participant and their doctor will know which treatment is received. During the study the participant will receive MBX-8025 (study medication) in a 5 mg, 10 mg or 25 mg dose. There will be no placebo (pill without active ingredient)
The participation in this study will last a maximum of 224 days (32 weeks): A screening period (up to 2 weeks), an open-label initial treatment period (8 weeks), an open-label extension period (18 weeks) and a follow-up period (4 weeks) or early termination visit.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
16/WM/0471
Date of REC Opinion
23 Dec 2016
REC opinion
Further Information Favourable Opinion