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Recruiting NCT04645251

Polycystic Liver Disease Registry (UK)

Observational Polycystic Liver Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Polycystic Liver Disease. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Netherlands, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Overview

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

Detailed description

The Polycystic Liver Disease (PLD) registry (UK) is the UK specific part of an international, multicentre, observational registry. The overall international PLD registry is led by Radboud University Medical Center, Nijmegan, Netherlands. Note- please direct any queries about the international registry to the team at Radboud University Medical Center.

All patients with Polycystic Kidney Disease (PKD) with PLD or patients with Isolated (without PKD) Polycystic Liver Disease are eligible for inclusion (\>10 liver cysts).

Data will be collected prospectively and retrospectively including a specific validated PLD questionnaire (PLD-Q).

This registry provides us insight in patient characteristics, risk factors, symptoms, quality of life and treatment strategies in the biggest international PLD cohort so far. Results of this registry will be published and shared at national and international congresses.

Primary outcome measures

  • Map the natural course of PLD [Time frame: 10 years]
Secondary outcome measures (5)
  • Assess quality of life using the validated PLD questionnaire (PLD-Q) [Time frame: 10 years]
  • Elucidate risk factors for developing a complication in PLD [Time frame: 10 years]
  • Elucidate elements that affect PLD progression [Time frame: 10 years]
  • Compare effectiveness of different therapies [Time frame: 10 years]
  • Determine whether rate of liver growth can be used to predict disease progression [Time frame: 10 years]

Eligibility criteria

Inclusion criteria

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

Exclusion criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Observational model
Cohort

Study locations

Netherlands · 1 center
  • Radboud University Medical Center — Nijmegen
United Kingdom · 1 center
  • Portmouth Hospitals University NHS Trust — Portsmouth

Publications

  • 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
  • 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
  • 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

Identifiers

NCT: NCT04645251 · 228220

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗