A study to evaluate the effect of Obeticholic Acid to treat patients with a recent Primary Biliary Cholangitis (PBC) diagnosis who also experience issues with cognitive function around memory and problem solving

Not Applicable

• Conditions: Primary biliary cholangitis (PBC) with cognitive symptoms around memory and problem solving; Digestive System; Primary biliary cirrhosis

• Registration Number: ISRCTN45490220
• Lead Sponsor: ewcastle upon Tyne Hospitals NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-08-02
• Last Update Posted: 29 April 2024
• Study Completion: 2025-02-28

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

1. Established diagnosis of PBC based on the presence of 2 out of the 3 key disease characteristics:
1.1. AMA or PBC-specific ANA at a titre of 1/40 or greater
1.2. Elevated alkaline phosphatase (above the upper limit of normal for the relevant laboratory)
1.3. Compatible or diagnostic liver biopsy
2. Diagnosed disease duration of <2 years
3. PBC-40 Cognitive Domain score of =12 at screening
4. Stable UDCA dose for 3 months (either at 13-15 mg/kg) or not on UDCA if intolerant
5. Willing to complete the study assessment protocols
6. For participants of childbearing potential: willing to use highly effective contraception or to practice true abstinence to avoid pregnancy for the entire duration of the treatment period
7. Good command of the English language (to ensure that participants are able to comply with cognitive testing)
8. Ability to consent, to comply with the study protocol, and to attend clinic visits
9. Aged =18 and =65 years

Exclusion Criteria

1. Clinical suspicion of advanced disease evidenced by a history of one or more of the following:
1.1. Ascites requiring diuretic therapy or percutaneous drainage
1.2. Endoscopically confirmed varices
1.3. Liver biopsy suggesting cirrhosis
1.4. Platelet count <150
1.5. Bipolar spleen length >12 cm on ultrasound
1.6. Hepatocellular carcinoma conformed by biopsy or 2 imaging modalities
2. Bilirubin >1.5 x upper limit of normal (ULN)
3. Complete biliary obstruction
4. Fibroscan >17.6 kPa within the year prior to or at screening
5. Inter-current disease characterised by cognitive dysfunction (e.g. dementia or neurodegenerative disease) or clinical suspicion of age-related cognitive decline
6. Inter-current medication characterised by cognitive dysfunction (benzodiazepines, opiates other than codeine phosphate, sleeping pills, anti-psychotic agents, regular daily anti-histamine use in the last four weeks, or recreational drug use).
7. Anticipated change in PBC medication within the duration of the study
8. Contraindications to contrast free MRI assessment (active medical implants such as Cardiac pacemaker or metal implants)
9. Previous exposure to OCA (either in clinical trials or in clinical practice) or Fibrate therapy for =3 months and within the last 3 months
10. Regular (more than one week per month) alcohol consumption in excess of recommended safe limits (14 units per week)
11. Active participation in another interventional trial or exposure to another experimental drug within 5 half-lives
12. Pregnancy or planning to get pregnant
13. Clinical diagnosis of AIH overlap
14. Concurrent liver disease of another aetiology
15. Severe pruritus (>11 on PBC-40 pruritus domain)
16. Hypersensitivity to the active substance or to any of the excipients
17. Treating clinician deems the patient is not suitable to participate in the trial based on other criteria apparent during screening or from medical history

Study & Design

• Study Type: Interventional
• Study Design: Not specified