Polycystic Liver Disease Registry (UK)

NCT04645251 | Recruiting DMC

• 1 other identifier: 228220
• Study Type: observational
• Target: 500
• Locations: 2 countries
• Sites: 2

Brief Summary

Multicentre, observational registry studying the natural course of polycystic liver disease.

Conditions

Polycystic Liver Disease

Outcome Measures

Primary Outcomes (1)

• Map the natural course of PLD

  To establish the demographics, presence and severity of symptoms (quantified using the validated PLD-Q questionnaire), liver cyst characteristics on imaging (assessed using Qian's classification), biochemical marker abnormalities (including gamma-glutamyl transferase and alkaline phosphatase), rate and nature of complications, need for treatment, change in liver volume in patients with PLD. This data will be collected retrospectively and prospectively from clinical records and the PLD-Q questionnaire.

  10 years

Secondary Outcomes (5)

• Assess quality of life using the validated PLD questionnaire (PLD-Q)

  10 years

• Elucidate risk factors for developing a complication in PLD

  10 years

• Elucidate elements that affect PLD progression

  10 years

• Compare effectiveness of different therapies

  10 years

• Determine whether rate of liver growth can be used to predict disease progression

  10 years

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Mixture of adult patients with PKD and PLD

You may qualify if:

• Patients with PLD with more than 10 liver cysts
• Patients with PKD and PLD with more than 10 liver cysts

You may not qualify if:

• Patients with PKD or PLD with less than 10 liver cysts

Sponsors & Collaborators

• Portsmouth Hospitals NHS Trust: lead
• Radboud University Medical Center: collaborator

Study Sites (2)

Radboud University Medical Center

Nijmegen, 6525, Netherlands

RECRUITING

Portmouth Hospitals University NHS Trust

Portsmouth, Hampshire, PO63LY, United Kingdom

RECRUITING

Related Publications (3)

• Neijenhuis MK, Gevers TJ, Hogan MC, Kamath PS, Wijnands TF, van den Ouweland RC, Edwards ME, Sloan JA, Kievit W, Drenth JP. Development and Validation of a Disease-Specific Questionnaire to Assess Patient-Reported Symptoms in Polycystic Liver Disease. Hepatology. 2016 Jul;64(1):151-60. doi: 10.1002/hep.28545. Epub 2016 Apr 15.

  PMID: 26970415 BACKGROUND

• van Aerts RMM, van de Laarschot LFM, Banales JM, Drenth JPH. Clinical management of polycystic liver disease. J Hepatol. 2018 Apr;68(4):827-837. doi: 10.1016/j.jhep.2017.11.024. Epub 2017 Nov 24.

  PMID: 29175241 BACKGROUND

• Qian Q, Li A, King BF, Kamath PS, Lager DJ, Huston J 3rd, Shub C, Davila S, Somlo S, Torres VE. Clinical profile of autosomal dominant polycystic liver disease. Hepatology. 2003 Jan;37(1):164-71. doi: 10.1053/jhep.2003.50006.

  PMID: 12500201 BACKGROUND

MeSH Terms

Conditions

Polycystic liver disease

Central Study Contacts

Richard Aspinall, BSc, MBChB, PhD

CONTACT

02392286255; richard.aspinall@porthosp.nhs.uk

Benjamin Giles, BSc, MBBS

CONTACT

02392286000; benjamin.giles@porthosp.nhs.uk

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 10 Years
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 11, 2020
• First Posted: November 27, 2020
• Study Start: January 21, 2021
• Primary Completion (Estimated): December 1, 2030
• Study Completion (Estimated): December 1, 2030
• Last Updated: December 23, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR
• Time Frame: Anonymised data will be stored for 15 years

Locations

NL (1); GB (1)