Polycystic Liver Disease Registry (UK)

Polycystic Liver Disease Clinical Trial

— PLD  

Official title:

Polycystic Liver Disease Registry (UK)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04645251
• Other study ID #: 228220
• Status: Recruiting
• Start date: January 21, 2021
• Est. completion date: December 1, 2030

Study information

• Verified date: April 2023
• Source: Portsmouth Hospitals NHS Trust
• Contact: Richard Aspinall, BSc, MBChB, PhD
• Phone: 02392286255
• Email: richard.aspinall[@]porthosp.nhs.uk
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: December 1, 2030
• Est. primary completion date: December 1, 2030
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cysts
• Liver Diseases
• Polycystic Liver Disease

Locations

Country	Name	City	State
Netherlands	Radboud University Medical Center	Nijmegen
United Kingdom	Portmouth Hospitals University NHS Trust	Portsmouth	Hampshire

Sponsors (2)

Lead Sponsor	Collaborator
Portsmouth Hospitals NHS Trust	Radboud University Medical Center

Countries where clinical trial is conducted

Netherlands,  United Kingdom, 

References & 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. — View 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. — View 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Assess quality of life using the validated PLD questionnaire (PLD-Q)	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.	10 years
Secondary	Elucidate risk factors for developing a complication in PLD	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).	10 years
Secondary	Elucidate elements that affect PLD progression	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).	10 years
Secondary	Compare effectiveness of different therapies	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.	10 years
Secondary	Determine whether rate of liver growth can be used to predict disease progression	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	10 years