Phase 2 EDP-305 in Patients with Primary Biliary Cholangitis
Research type
Research Study
Full title
A PHASE 2 DOSE RANGING, RANDOMIZED, DOUBLE BLIND, PLACEBO-CONTROLLED STUDY EVALUATING THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF EDP-305 IN SUBJECTS WITH PRIMARY BILIARY CHOLANGITIS (PBC) WITH OR WITHOUT AN INADEQUATE RESPONSE TO URSODEOXYCHOLIC ACID (UDCA)
IRAS ID
240366
Contact name
Michael Heneghan
Contact email
Sponsor organisation
Enanta Pharmaceuticals, Inc.
Eudract number
2017-003528-62
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
Bile is a liquid produced inside the liver that's used to help digest fats and remove waste products from the body. It passes out of the liver through small tubes called bile ducts.
In Primary biliary cholangitis (PBC), the immune system (the body's natural defence against infection and illness) mistakenly attacks the bile ducts.
The bile ducts become damaged and injured, causing bile to build up in the liver. This further damages the liver (fibrosis) and may lead to scarring (cirrhosis).
There is only one approved treatment for PBC, with ursodeoxycholic acid (UDCA). UDCA is not effective in up to 40% of patients.There remains an unmet need for better therapies in patients with an inadequate response to UDCA, those who cannot tolerate it and those with a more advanced fibrosis stage.
FXR is a nuclear hormone receptor (a protein in the cell that regulates bile acid activity) that when activated starts a process that contributes to a reduction in the bile acid pool size.
EDP-305 is an FXR agonist (meaning it will combine and ‘switch on’ the FXR receptor).Early study results suggest that EDP_305 has the potential to satisfy the unmet medical need.
This study will look at EDP-305 in approximately 119 participants, who will be in the study for about 20 weeks, visiting the study site 8 times. Participants will take the study medication tablets once daily for 12 weeks.
Participants will be randomly assigned to take 1mg EDP-305, 2.5mg EDP-305 or placebo (a tablet that looks exactly like EDP-305 but has no active ingredient). The study will be double-blind which means that neither the participant nor the study team will know which medication the participant is taking.
Throughout the study, participants will have study visits for safety monitoring and additional examinations (including blood sampling).
REC name
Wales REC 2
REC reference
18/WA/0211
Date of REC Opinion
27 Jul 2018
REC opinion
Further Information Favourable Opinion