Development and Assessment of The Polycystic Liver Disease Questionnaire (PLD-Q).

NCT02173080 | Completed

• 1 other identifier: 14-003832
• Study Type: observational
• Target: 594
• Locations: 1 country
• Sites: 1

Brief Summary

This Pilot study will enable development \& assessment of a Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q). Polycystic liver disease (PLD) is characterized by the formation of numerous cysts in the liver, and can lead to severe symptomatic hepatomegaly. It is common in patients with autosomal dominant polycystic liver disease (ADPLD) and autosomal dominant polycystic kidney disease (ADPKD).

Conditions

Polycystic Liver Disease

Keywords

autosomal dominant polycystic liver disease; autosomal dominant polycystic kidney disease; quality of life; patient reported outcome tool

Outcome Measures

Primary Outcomes (1)

• Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q) total score

  total score on the PLD-Q

  baseline to 6 months

Secondary Outcomes (5)

• The Short Form (36) Health Survey (SF36)

  baseline to 6 months

• European Organisation for Research and Treatment of Cancer Quality of life questionnaire (EORTC-QLQ-C30) symptom subscale

  baseline to 6 months

• Euroqol group 5 dimensions questionnaire visual analog score (EQ5D-VAS)

  baseline to 6 months

• Total liver volume

  baseline

• Total kidney volume

  baseline

Study Arms (5)

Polycystic liver disease patients

will receive PLD-Q, EORTC QLQ-30 symptoms subscale, EQ5D-VAS score and SF36

Other: PLD-Q

ADPKD group without PLD

will receive PLD-Q

Healthy controls

receive PLD-Q

Other: PLD-Q

PLD patient focus group

to discuss and improve PLD-Q

PLD clinical expert focus group

to discuss and improve PLD-Q

Interventions

PLD-Q OTHER

polycystic liver disease questionnaire

Healthy controls; Polycystic liver disease patients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with polycystic liver disease

You may qualify if:

• Patients ≥ 18 year
• Polycystic liver, defined by \> 20 liver cysts on imaging

You may not qualify if:

• Patients unable to speak or read the English properly
• History of kidney or liver transplantation
• Dialysis
• Current use of experimental drugs (e.g. lanreotide, octreotide)
• Recent liver resection or major surgery
• Other comorbidities that can affect the outcome of the questionnaire, as deemed by the investigators

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Polycystic liver disease; Polycystic Kidney, Autosomal Dominant

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Study Officials

• Marie C Hogan

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PI

Study Record Dates

• First Submitted: June 20, 2014
• First Posted: June 24, 2014
• Study Start: June 1, 2014
• Primary Completion: March 11, 2024
• Study Completion: March 11, 2024
• Last Updated: September 19, 2024

Record last verified: 2024-09

Locations

US (1)