Extension study of Seladelpar in PBC
Research type
Research Study
Full title
An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Seladelpar in Subjects with Primary Biliary Cholangitis (PBC)
IRAS ID
238195
Contact name
Douglas Thorburn
Contact email
Sponsor organisation
CymaBay Therapeutics, Inc.
Eudract number
2017-003910-16
Duration of Study in the UK
2 years, 4 months, 17 days
Research summary
This is a phase 2/3 open label, long-term study evaluating the safety and tolerability of seladelpar in patients with Primary Biliary Cholangitis (PBC).
PBC is a serious and potentially life threatening autoimmune disease of the liver characterised by impaired bile flow and accumulation of toxic bile acids. Clinical symptoms of PBC include fatigue and pruritus (itchy skin), which can be quite disabling. PBC is characterised by liver inflammation and immune-mediated destruction of bile ducts, which leads to decreased bile secretion and the retention of toxic substances within the liver, resulting in further liver damage, fibrosis, cirrhosis and eventually liver failure.
The study medication works by targeting and initiating a response from a particular protein in the body which is involved in lipid processing in the liver and muscle. The study medication has shown in laboratory and previous human studies that it might be useful in treating the signs and symptoms of certain lipid disorders. It is believed, that PBC patients may also potentially benefit from this new study medication as PBC is closely linked to lipid metabolism.
Participants who successfully completed a previous study with seladelpar will be invited to continue treatment in this long-term safety study, ideally to follow on from the previous study allowing continuous treatment. Up to 128 participants at sites in Europe and North America will be enrolled. This is an open label study meaning that participants and their doctors will know which treatment is received. During the study participants will receive an oral seladelpar l 1, 2, 5, or 10 mg dose once daily, made up of 1, 5, 10 mg capsules. Treatment will continue until seladelpar is commercially available or the program is discontinued. Participants will have a site visit every three months (90 days). During the treatment period, the dose can be adjusted for safety or efficacy reasons.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
18/SC/0009
Date of REC Opinion
8 Feb 2018
REC opinion
Further Information Favourable Opinion