Rare disease research can be challenging and often there are no precedents to rely on. This case study looks at the intelligent recommendations and actions implemented by Cmed for a Phase II trial for a promising new drug with a novel mechanism of action to treat the progressive Primary Biliary Cholangitis (PBC) disease.
Cmed is the right partner to help navigate hidden challenges, maximize outcomes, and deliver on time and within budget because of their depth of experience and operational expertise.
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. At this stage, the disease progresses due to chronic cholestasis, secondary inflammation and fibrosis, and may even lead to liver cirrhosis and liver failure.
The reported prevalence of PBC ranges from 20-400 cases per million of the population and is more common in women than men (9:1 ratio) and mostly diagnosed between the ages of 30 and 60 years.
Other complications of PBC include hepatocellular carcinoma, metabolic bone disease, and malabsorption. PBC is a progressive disease in most patients. It eventually becomes irreversible, and therefore untreatable.
Current management of patients with PBC consists of facilitation of bile flow coupled with management of its symptoms and complications. This however does not address the underlying disease. The only widely accepted treatment is Ursodeoxycholic Acid (UDCA), which can delay disease progression and improve long-term survival.
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 has worked on over 100 clinical trials for rare diseases. Talk to us about how we can help with your next trial. Email us at email@example.com