ENHANCE: A Study of Primary Biliary Cholangitis
Research type
Research Study
Full title
A 52-week, placebo-controlled, randomized, Phase 3 study to evaluate the safety and efficacy of seladelpar in subjects with primary biliary cholangitis (PBC) and an inadequate response to or an intolerance to ursodeoxycholic acid (UDCA)
IRAS ID
251717
Contact name
David Jones
Contact email
Sponsor organisation
CymaBay Therapeutics, Inc.
Eudract number
2018-001171-20
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 9 months, 14 days
Research summary
Primary biliary cholangitis (PBC) is a disease which causes destruction of bile ducts (tubes which carry bile) in the liver and causes build-up of toxic bile acids (BA). This build-up is known as cholestasis and is one of the main characteristics of PBC. Increase of can lead to fibrosis, cirrhosis and eventually liver failure. The most common symptoms of PBC are fatigue (feeling tired) and pruritus (itching). Successful treatment of PBC reduces the build-up of the in the liver and its associated symptoms. First treatment option for PBC is ursodeoxycholic acid; (UDCA) however, up to 40% of patients are considered inadequate responders. These patients can be treated with obeticholic acid, but some patients do not respond adequately or are intolerant to this medication. Therefore, additional treatments are needed for the long-term treatment of PBC.
The drug being tested is seladelpar. In previous human studies, seladelpar decreased cholestasis, improved pruritus and has been well tolerated. The purpose of this study is to evaluate the safety and efficacy of seladelpar in patients with PBC and an inadequate response or intolerance to UDCA.
Patients will have a one-in-three chance of receiving seladelpar 5 mg, seladelpar 10 mg, or placebo once per day dosing. If participants enter the study on UDCA they will continue this throughout. The study consists of a 2-week screening period, 2-week run-in period and a 52-week treatment period. Once the participants have completed the treatment period, they will attend a follow up visit 4 weeks after their last dose or enter a long-term safety study.
Procedures involved include, physical exams, vital signs, blood samples, ECGs, questionnaire completion, liver elastography and optional liver biopsy. The study is sponsored by CymaBay Therapeutics, Inc. Approximately 240 patients will take part globally with approximately 25 patients in the UK.
REC name
West of Scotland REC 1
REC reference
18/WS/0201
Date of REC Opinion
17 Dec 2018
REC opinion
Further Information Favourable Opinion