What the trial was testing
The RESPONSE enrolled 193 patients with primary biliary cholangitis. The study was sponsored by Gilead Sciences and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
62% had a biochemical response on seladelpar vs. 20% on the comparison pill.
New England Journal of Medicine · 2024 · NCT04620733
These findings — that biochemical response at 12 months on seladelpar plus ursodeoxycholic acid — were published in the New England Journal of Medicine and represent the headline result of the study.
Researchers tracked outcomes across 193 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with primary biliary cholangitis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Seladelpar (Livdelzi) is FDA-approved and available now for primary biliary cholangitis when ursodeoxycholic acid is not enough. It also reduced the moderate-to-severe itching that drives many PBC patients up the wall. Ask a hepatologist about adding it to your existing regimen.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open primary biliary cholangitis trials
A-LiNK: Improving Outcomes in Autoimmune Liver Disease
The Autoimmune Liver disease Network for Kids (A-LiNK) is a multi-institutional group with the mission to deliver the best care to kids with pediatric autoimmune liver disease (AILD). This study will establish a shared clinical registry and a learning health network for the participating sites focusing on collecting and transmitting clinical measurement data, information about processes, and participation in an improvement collaborative. Pediatric Autoimmune Hepatitis (AIH) and Primary Sclerosing Cholangitis (PSC), represent a spectrum of AILD which present unique diagnostic and therapeutic challenges.A lack of accepted guidelines for disease monitoring or symptom management results in wide treatment variation with liver transplants indicated in refractory, progressive disease. The aims of A-LiNK are to: 1.) Create a learning health network focused on patient-centered outcomes research characterized by transparent sharing among centers, common priorities, and feasible plans for implementing new practices; 2) shift from traditional investigator-driven study to a patient and family-centered approach, and 3.) improve clinical outcomes and quality of life for pediatric AILD patients.
National Database on Primary Biliary Cholangitis
Primary biliary cholangitis (PBC) is a rare, autoimmune, cholestatic liver disease. No data about the disease epidemiology exist in Italy. Therefore this study aims to develop a national PBC patient database linked to a biological sample storage.