RESPONSE
Research type
Research Study
Full title
RESPONSE: A Placebo-controlled, Randomized, Phase 3 Study to Evaluate the Efficacy and Safety of Seladelpar in Patients With Primary Biliary Cholangitis (PBC) and an Inadequate Response to or an Intolerance to Ursodeoxycholic Acid (UDCA)
IRAS ID
289007
Contact name
David Jones
Contact email
Sponsor organisation
CymaBay Therapeutics, Inc.
Eudract number
2020-004348-27
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 6 months, 4 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. 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. Other studies in PBC patients have shown that seladelpar may be useful for lowering alkaline phosphatase (ALP) levels and decreasing itching. Seladelpar has been tested previously in approximately 16 clinical studies and the results have been generally safe and well tolerated.
The purpose of this study, is to determine if seladelpar, is safe and effective in treating patients with PBC compared to placebo (which looks like the study drug but is not active medication).
This is a randomised, double-blind and placebo-controlled study. Randomised means participants will be selected by chance to receive either seladelpar or placebo. Double-blinded refers to both the participant and the study doctor not knowing if the participant is taking seladelpar or placebo.
Patients will have a two-in-three chance of receiving seladelpar 10 mg, or placebo once per day
dosing. If participants enter the study on UDCA they will continue this throughout. In a blinded fashion, study treatment may be decreased to a lower dose if deemed necessary for safety or tolerability reasons. This means that participants on 10 mg seladelpar may be decreased to 5 mg.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, urine tests, ECGs, questionnaire completion, fibroscan, abdominal ultrasound and optional liver biopsy. The study is sponsored by CymaBay Therapeutics, Inc. Approximately 180 patients will take part globally with approximately 20 patients in the UK.
REC name
London - Surrey Borders Research Ethics Committee
REC reference
21/LO/0172
Date of REC Opinion
11 May 2021
REC opinion
Further Information Favourable Opinion