Phase 2 - MBX-8025 in Participants with Primary Biliary Cirrhosis
Research type
Research Study
Full title
A 12-week, double-blind, randomized, placebo-controlled, Phase 2 study, to evaluate the effects of two doses of MBX-8025 in subjects with Primary Biliary Cirrhosis (PBC) and inadequate response to ursodeoxycholic acid (UDCA)
IRAS ID
185803
Contact name
Gideon Hirschfield
Contact email
Sponsor organisation
CymaBay Therapeutics, Inc.
Eudract number
2015-002698-39
Duration of Study in the UK
0 years, 9 months, 9 days
Research summary
The purpose of this clinical study is to establish the effects of two different dose levels of a new study medication, called MBX-8025, in participants with Primary Biliary Cirrhosis (PBC) who are already taking ursodeoxycholic acid (UDCA).
PBC is a slowly progressive autoimmune disease (where the body attacks its own healthy cells and tissue) of the liver that mainly affects women. It is a long-term, life-threatening disease which can lead to cirrhosis (scarring) of the liver, liver failure and may increase the risk of liver cancer.
MBX-8025 has shown potential at decreasing the cholestasis that occurs in PBC. Cholestasis is where bile - the digestive liquid produced in your liver - cannot flow from the liver to the small intestine. MBX-8025 works by increasing bile flow and decreasing the production of bile acids. It is also thought to reduce the PBC-associated inflammation.
Male and female PBC patients, aged between 18-75, who have been on UDCA treatment for six months, are eligible to take part in this study.
Approximately 75 participants will be enrolled into the study at approximately 14 research sites across the United Kingdom, Norway, Netherlands, Canada and the United States.
Participants will be randomised (like drawing numbers from a hat) to receive either 50 mg or 200 mg of MBX-8025, or a placebo (an inactive substance that looks like the study medication but contains no medication) dose of MBX-8025. For 12 weeks, they will receive one dose per day in the form of capsules to be taken orally. Participants will visit the clinic 5 times during the treatment period.
Participants will undergo various procedures at each clinic visit including physical examinations, vital sign measurements, blood samples, electrocardiograms, liver imaging scans, itching assessments and quality of life questionnaires.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
15/WM/0364
Date of REC Opinion
3 Nov 2015
REC opinion
Further Information Favourable Opinion