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Polycystic Liver Disease Registry (UK) (PLD)

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ClinicalTrials.gov Identifier: NCT04645251
Recruitment Status : Recruiting
First Posted : November 27, 2020
Last Update Posted : June 11, 2021
Sponsor:
Collaborator:
Radboud University Medical Center
Information provided by (Responsible Party):
Portsmouth Hospitals NHS Trust

Study Description
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Brief Summary:
Multicentre, observational registry studying the natural course of polycystic liver disease.

Condition or disease
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.

Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 500 participants
Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration: 10 Years
Official Title: Polycystic Liver Disease Registry (UK)
Actual Study Start Date : January 21, 2021
Estimated Primary Completion Date : December 1, 2030
Estimated Study Completion Date : December 1, 2030

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases

Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Map the natural course of PLD [ Time Frame: 10 years ]
    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.


Secondary Outcome Measures :
  1. Assess quality of life using the validated PLD questionnaire (PLD-Q) [ Time Frame: 10 years ]
    The PLD-Q questionnaire consists of 18 items assessing symptom frequency and severity. It gives a score (0-100) based on the patient's answers with a higher score indicating more severe, symptomatic disease with a poorer quality of life.

  2. Elucidate risk factors for developing a complication in PLD [ Time Frame: 10 years ]
    For patients that develop a complication of PLD (including jaundice, abdominal hernia, features of portal hypertension, cyst haemorrhage and cyst infection) we will look through their clinical record to establish if there any risk factors which are common to those with a complication (e.g. age, gender, medications, cyst phenotype).

  3. Elucidate elements that affect PLD progression [ Time Frame: 10 years ]
    For patients that have a worsening of their PLD (based on increasing symptoms as per the PLD-Q questionnaire, development of complications or need for treatment) we will look through their clinical records to establish if there any elements which are common to those with progressive disease (e.g. age, gender, medications).

  4. Compare effectiveness of different therapies [ Time Frame: 10 years ]
    For patients who have had or require treatment for PLD we will compare the effectiveness of that treatment based on change in symptoms (based on PLD-Q questionnaire), recurrence of symptoms, development of complications or need for further treatment.

  5. Determine whether rate of liver growth can be used to predict disease progression [ Time Frame: 10 years ]
    Liver growth rate will be determined using change in liver volume on serial radiology imaging. This will be compared against patients symptoms (measured via PLD-Q questionnaire), development of complications and need for treatment to establish whether there is a correlation in rate of liver growth and disease progression


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Mixture of adult patients with PKD and PLD
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04645251


Contacts
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Contact: Richard Aspinall, BSc, MBChB, PhD 02392286255 richard.aspinall@porthosp.nhs.uk
Contact: Benjamin Giles, BSc, MBBS 02392286000 ext 3154 benjamin.giles@porthosp.nhs.uk

Locations
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Netherlands
Radboud University Medical Center Recruiting
Nijmegen, Netherlands, 6525
Contact: Thijs RM Barten       thijs.rm.barten@radboudumc.nl   
Contact: Joost PH Drenth       joostphdrenth@cs.com   
United Kingdom
Portmouth Hospitals University NHS Trust Recruiting
Portsmouth, Hampshire, United Kingdom, PO63LY
Contact: Benjamin Giles       benjamin.giles@porthosp.nhs.uk   
Contact: Richard Aspinall       richard.aspinall@porthosp.nhs.uk   
Sponsors and Collaborators
Portsmouth Hospitals NHS Trust
Radboud University Medical Center
More Information
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Publications:

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Responsible Party: Portsmouth Hospitals NHS Trust
ClinicalTrials.gov Identifier: NCT04645251    
Other Study ID Numbers: 228220
First Posted: November 27, 2020    Key Record Dates
Last Update Posted: June 11, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Anonymised participant data will be shared with research team at Radboud University Medical Center, Nijmegen, Netherlands.
Supporting Materials: Study Protocol
Clinical Study Report (CSR)
Time Frame: Anonymised data will be stored for 15 years

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cysts
Liver Diseases
Digestive System Diseases
Neoplasms
Pathological Conditions, Anatomical