Clinical trial NCT04645251: Polycystic Liver Disease Registry (UK)
Official title: Polycystic Liver Disease Registry (UK)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Polycystic Liver Disease
Lead sponsor: Portsmouth Hospitals NHS Trust
Sponsor class: OTHER_GOV
Collaborators: name: Radboud University Medical Center
First posted: 2020-11-27
Enrollment: 500
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Mixture of adult patients with PKD and PLD

Brief summary: 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.

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

Primary outcomes:
measure: Map the natural course of PLD
timeFrame: 10 years
Secondary outcomes:
measure: Assess quality of life using the validated PLD questionnaire (PLD-Q)
timeFrame: 10 years
measure: Elucidate risk factors for developing a complication in PLD
timeFrame: 10 years
measure: Elucidate elements that affect PLD progression
timeFrame: 10 years
measure: Compare effectiveness of different therapies
timeFrame: 10 years
measure: Determine whether rate of liver growth can be used to predict disease progression
timeFrame: 10 years
Other outcomes:
Locations:
city: Nijmegen
country: Netherlands
facility: Radboud University Medical Center
city: Portsmouth
country: United Kingdom
facility: Portmouth Hospitals University NHS Trust
state: Hampshire
Study officials:
References:
citation: 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.
citation: 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.
citation: 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.