Clinical Study to Assess Markers of Disease Activity in Patients with PBC
Research type
Research Study
Full title
"A Clinical Study to Assess Biomarkers of Disease Activity and Patient Reported Outcome Measures in Patients with Primary Biliary Cirrhosis"
IRAS ID
171199
Contact name
Jelena Mijatovic
Contact email
Sponsor organisation
Novartis Pharma AG
Duration of Study in the UK
0 years, 4 months, 1 days
Research summary
Primary Biliary Cirrhosis (PBC) is a relatively uncommon chronic disorder affecting the small tubes in the liver responsible for transporting bile to the intestine. As these ducts become damaged, a build-up of bile develops and causes inflammation and scarring to liver tissue. This scarring reduces the liver's ability to remove toxins from the blood and leads to extreme tiredness, skin itching, and eventually jaundice and liver failure. About 9 out of 10 people affected by this disease are women, and the initial trigger is as yet unknown.
Because many patients with PBC respond well to treatment with the bile acid, ursodeoxycholic acid (UDCA), timely diagnosis is key to a successful outcome. Blood tests for specific antibodies and soluble markers, together with ultrasound imaging of the liver, and Patient Reported Outcomes (PROs) are used to confirm PBC and a better understanding of these non-invasive techniques will lead to earlier diagnosis without the need for liver biopsy at a later stage in the disease process.
This is a non-interventional, multi-centre UK study and will evaluate performance and variability of biomarkers for PBC in a total of 30 adults. Following an initial screening, two further visits will take place within approx. 30 days. Participants will also be required to complete a skin itch assessment using Visual Analogue Scale (VAS) and PBC-40 questionnaires at designated times between visits 2 and 3.
In addition, the study will include an optional pharmacogenetics/genomics component involving DNA extraction from a blood sample taken at visit 2. This will explore genetic links with PBC progress and may assist identification of other new bio-markers.REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
15/NE/0194
Date of REC Opinion
3 Aug 2015
REC opinion
Further Information Favourable Opinion