IDEAL study aims to determine the impact of seladelpar on alkaline phosphatase levels.
CymaBay Therapeutics – a company focusing on providing access to therapies for those with liver disease and other chronic diseases – has announced the initiation of its 52-week, placebo-controlled, phase 3 trial.
The IDEAL study aims to determine the impact of seladelpar on the normalisation of alkaline phosphatase (ALP) levels among subjects with primary biliary cholangitis (PBC).
The trial will ultimately enroll 75 patients with PBC who have experienced an incomplete response, or intolerance to, ursodeoxycholic acid. Patients will be randomly assigned using a 2:1 ratio to oral, once daily seladelpar 10 mg or placebo. The primary outcome measure is the normalisation at exactly one year.
Other vital outcomes evaluating efficacy include the percent change from baseline in ALP at 52 weeks and the level of self-reported itching at six months for those patients with moderate to severe symptoms at baseline.
Dr Cynthia Levy, Professor of Medicine at the University of Miami, believes the latest stage of the study could be vital: “Over the last few years there has been increasing interest in the medical community to understand how normalization of biochemical markers relates to risk of disease progression.”
She added: “The IDEAL study is an important step forward in assessing the potential of therapeutic intervention in normalising biochemical markers of PBC in patients with incomplete control of ALP levels. Our hope is that the findings from this research will help advance scientific understanding of the role of ALP normalization in PBC, particularly when it comes to addressing some of the most distressing symptoms for patients.”
Charles McWherter, Chief Scientific Officer and President of Research and Development at CymaBay concluded: “We see the IDEAL study as an opportunity to evaluate the potential benefits of seladelpar in a population that has a persistent elevation of ALP, but whose ALP levels are not above the 1.67 times upper-limit-of-normal recommended by clinical guidelines or that are used for entering clinical trials of new second-line agents.”
PBC remains a rare, chronic inflammatory liver disease that is most prevalent among women, with one in 1,000 over the age of 40 affected. It is defined by impaired bile flow and the build-up of toxic bile acids in the liver.