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

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ClinicalTrials.gov Identifier: NCT02173080
Recruitment Status : Unknown
Verified April 2015 by Marie Hogan, Mayo Clinic.
Recruitment status was:  Recruiting
First Posted : June 24, 2014
Last Update Posted : April 7, 2015
Information provided by (Responsible Party):
Marie Hogan, Mayo Clinic

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).

Condition or disease Intervention/treatment
Polycystic Liver Disease Other: PLD-Q

Detailed Description:

As liver function is preserved, quality of life is the main outcome in PLD. Patient reported outcomes are increasingly utilized to assess treatment efficacy & can include any endpoint derived from patient reports & are usually assessed by questionnaire. Several existing generic questionnaires assess gastrointestinal symptoms, but include irrelevant items for polycystic liver disease (PLD) & do not examine relevant extra-abdominal symptoms. Also, a questionnaire that assesses the full range of PLD related problems is more likely to be responsive to changes after treatment initiation. Therefore, a tool that accurately detects changes in PLD symptoms is required.

The development and first steps of validation of this questionnaire are already completed in Netherlands. We will further develop this questionnaire for English speaking patients and validate it in Mayo Clinic cohorts.

The study contains three phases. Phase 1: pilot testing of the directly translated questionnaire (from dutch into english) in a small group of polycystic liver disease (PLD) patients. We will include both symptomatic & asymptomatic patients (i.e. from mild to severe PLD). Phase 2: further refinement of the questionnaire using the results of the pilot test and input of Mayo patient and clinician focus groups. Phase 3: Further validation in a large cohort of 200 PLD patients with (1) comparison with 200 controls without PLD, (2) cross-sectional correlation with total liver (TLV) & kidney volumes (TKV), (3) comparing ADPKD patients with & without PLD (5) against other validated questionnaires (e.g. SF36) & (5) development of a custom "Worry Questionnaire" examining disease-related worries & concerns. Finally, longitudinal analyses will be performed to assess PLD-Q sensitivity to change in symptoms. Statistical & psychometric analyses will be provided by Mayo Clinic quality of life Research group, & developed in accordance with Food and Drug Administration (FDA) guidance on patient-reported outcome measures in consultation with their Study Endpoints & Labeling Development Division.

Study Type : Observational
Estimated Enrollment : 544 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Development and Assessment of the Psychometric Properties of a Polycystic Liver Disease-specific Patient Reported Outcomes Questionnaire (PLD-Q).
Study Start Date : June 2014
Estimated Primary Completion Date : June 2016
Estimated Study Completion Date : June 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases
U.S. FDA Resources

Group/Cohort Intervention/treatment
Polycystic liver disease patients
will receive PLD-Q, EORTC QLQ-30 symptoms subscale, EQ5D-VAS score and SF36
Other: PLD-Q
polycystic liver disease questionnaire
ADPKD group without PLD
will receive PLD-Q
Healthy controls
receive PLD-Q
Other: PLD-Q
polycystic liver disease questionnaire
PLD patient focus group
to discuss and improve PLD-Q
PLD clinical expert focus group
to discuss and improve PLD-Q

Primary Outcome Measures :
  1. Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q) total score [ Time Frame: baseline to 6 months ]
    total score on the PLD-Q

Secondary Outcome Measures :
  1. The Short Form (36) Health Survey (SF36) [ Time Frame: baseline to 6 months ]
  2. European Organisation for Research and Treatment of Cancer Quality of life questionnaire (EORTC-QLQ-C30) symptom subscale [ Time Frame: baseline to 6 months ]
  3. Euroqol group 5 dimensions questionnaire visual analog score (EQ5D-VAS) [ Time Frame: baseline to 6 months ]
  4. Total liver volume [ Time Frame: baseline ]
  5. Total kidney volume [ Time Frame: baseline ]
    measured in patients with autosomal dominant polycystic kidney disease (ADPKD)

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with polycystic liver disease

Inclusion Criteria:

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

Exclusion Criteria:

  • 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

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): NCT02173080

Contact: Marie C Hogan, MD, PhD 507-266-1963 Hogan.marie@mayo.edu

United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Marie C Hogan    507-266-1963    hogan.marie@mayo.edu   
Principal Investigator: Marie C Hogan         
Sub-Investigator: Tom Gevers         
Sub-Investigator: Patrick Kamath         
Sub-Investigator: Jeff Sloan         
Sub-Investigator: Joost Drenth         
Sponsors and Collaborators
Mayo Clinic
Principal Investigator: Marie C Hogan Mayo Clinic

Responsible Party: Marie Hogan, PI, Mayo Clinic
ClinicalTrials.gov Identifier: NCT02173080     History of Changes
Other Study ID Numbers: 14-003832
First Posted: June 24, 2014    Key Record Dates
Last Update Posted: April 7, 2015
Last Verified: April 2015

Keywords provided by Marie Hogan, Mayo Clinic:
autosomal dominant polycystic liver disease
autosomal dominant polycystic kidney disease
quality of life
patient reported outcome tool

Additional relevant MeSH terms:
Liver Diseases
Digestive System Diseases
Pathological Conditions, Anatomical