Optimising primary therapy in primary biliary cholangitis

Not Applicable

• Conditions: Primary biliary cholangitis; Digestive System

• Registration Number: ISRCTN17176388
• Lead Sponsor: ewcastle upon Tyne Hospitals NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-07-10
• Last Update Posted: 28 November 2023
• Study Completion: 2026-02-28

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 106

Inclusion Criteria

1. Established diagnosis of PBC based on the presence of at least 2 out of the 3 key disease characteristics, specifically:
1.1.AMA or PBC-specific ANA at a clinically diagnostic level
1.2.Elevated alkaline phosphatase (above the upper limit of normal (ULN) for the relevant laboratory)
1.3.Compatible or diagnostic liver biopsy
2. Ongoing elevation of alkaline phosphatase (=15% above ULN) at screening
3. Disease duration of <6 months from date of diagnosis at the time of consent
4. Use of UDCA for <3 months at the time of consent
5. Pre-treatment Ursodeoxycholic Acid Response Score with a predicted risk of future non-remission of liver biochemistry (ie ALP>ULN) with UDCA alone of >20%
6. For people of childbearing potential: an agreement to use at least an acceptable effective method contraception or to practise sexual abstinence to avoid pregnancy for entire duration of the study period.
7. Willing to complete the study assessment protocols
8. Ability to consent, able to comply with study protocol and attend clinic visits
9. Age = 18 years at the time of consent

Exclusion Criteria

1. Clinical contraindication to obeticholic acid use
2. Untreated, clinically significant pruritus (patients with effectively treated pruritus are eligible for inclusion)
3. Concomitant use of fibric acid derivatives (e.g. bezafibrate or fenofibrate) within 14 days prior to screening
4. Clinical suspicion of cirrhosis evidenced by a history of one or more of the following:
4.1. ascites requiring diuretic therapy or percutaneous drainage
4.2. endoscopically-confirmed varices
4.3. liver biopsy suggesting cirrhosis
4.4. platelet count <150 x109/L
4.5. transient elastography score >16.9 kPa within 3 months prior to or at screening
4.6. hepatocellular carcinoma confirmed by biopsy or 2 imaging modalities
4.7. hepatic encephalopathy
5. Bilirubin >twice the upper limit of normal
6. Evidence of complete biliary obstruction
7. Previous exposure to obeticholic acid (either in clinical trials or in clinical practice) or other potential PBC-modifying therapy
8. Regular (more than one week per month) alcohol consumption in excess of recommended safe limits (14 units per week)
9. Active participation in another interventional trial or exposure to another experimental drug within 5 half-lives
10. Pregnancy or planning to get pregnant within duration of participation in the trial
11. Currently breastfeeding
12. Overlapping features of an additional liver disease, including autoimmune hepatitis (using the Paris criteria for autoimmune hepatitis overlap)
13. Hypersensitivity to the active substance or to any of the excipients
14. If the participant’s treating clinician deems the patient is not suitable to participate in the trial based on other criteria apparent during screening or from medical history
15. Previous liver transplantation

Study & Design

• Study Type: Interventional
• Study Design: Not specified