Primary biliary cholangitis (PBC), previously called primary biliary cirrhosis, is a liver disease caused by auto-immune attack on bile duct cells leading to their gradual destruction and eventual disappearance.
PBC is a progressive disease in most patients and may lead to liver cirrhosis and failure. It eventually becomes irreversible, and therefore untreatable.
The only widely accepted treatment is Ursodeoxycholic Acid (UDCA), which can delay disease progression and improve long-term survival but does not address the underlying disease.
The response to UDCA, the standard therapy for PBC, is unfortunately not uniformly effective and many patients still experience significant toxicities. Therefore, there is an ongoing unmet need for improved and more well-tolerated therapies to address additional components of the disease.
The team at Cmed drew on their expertise to contribute to the study design and selection of appropriate endpoints for a Phase II trial with a European Pharmaceutical company.
Cmed helped with the response to the FDA feedback regarding selection of appropriate endpoints. As is often the case with rare disease research, there was no precedent. This was a new potential drug with a novel mechanism of action.
The trial successfully enrolled over 110 adult patients with PBC undergoing standard therapy. After a difficult start with slow enrolment, Cmed suggested changes to inclusion criteria, added sites who were better enrollers and recommended that the sponsor visit targeted sites to ensure their protocol was highly visible, as the drug was very promising.
As recruitment picked up, Operational teams were then able to focus on data capture and cleaning, leading to safety review assessments, as well as a key Interim analysis, final analysis (with top line results presented as soon as efficacy results were available), and clinical study report - all delivered on budget and within required timelines.
|Case Study Key Facts|
|Disease/indication||Primary Biliary Cholangitis|
|Type of study||Phase 2|
|Size of study||110 patients|