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

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by Mayo Clinic
Sponsor:
Information provided by (Responsible Party):
Marie Hogan, Mayo Clinic
ClinicalTrials.gov Identifier:
NCT02173080
First received: June 20, 2014
Last updated: NA
Last verified: June 2014
History: No changes posted
  Purpose

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 Intervention
Polycystic Liver Disease
Other: PLD-Q

Study Type: Observational
Study Design: 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).

Resource links provided by NLM:


Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q) total score [ Time Frame: baseline to 6 months ] [ Designated as safety issue: No ]
    total score on the PLD-Q


Secondary Outcome Measures:
  • The Short Form (36) Health Survey (SF36) [ Time Frame: baseline to 6 months ] [ Designated as safety issue: No ]
  • European Organisation for Research and Treatment of Cancer Quality of life questionnaire (EORTC-QLQ-C30) symptom subscale [ Time Frame: baseline to 6 months ] [ Designated as safety issue: No ]
  • Euroqol group 5 dimensions questionnaire visual analog score (EQ5D-VAS) [ Time Frame: baseline to 6 months ] [ Designated as safety issue: No ]
  • Total liver volume [ Time Frame: baseline ] [ Designated as safety issue: No ]
  • Total kidney volume [ Time Frame: baseline ] [ Designated as safety issue: No ]
    measured in patients with autosomal dominant polycystic kidney disease (ADPKD)


Estimated Enrollment: 544
Study Start Date: June 2014
Estimated Study Completion Date: June 2016
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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
autosomal dominant polycystic kidney disease without PLD
will receive PLD-Q
Other: PLD-Q
polycystic liver disease questionnaire
Healthy controls
will 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

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patients with polycystic liver disease

Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02173080

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

Locations
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
Investigators
Principal Investigator: Marie C Hogan Mayo Clinic
  More Information

No publications provided

Responsible Party: Marie Hogan, PI, Mayo Clinic
ClinicalTrials.gov Identifier: NCT02173080     History of Changes
Other Study ID Numbers: 14-003832
Study First Received: June 20, 2014
Last Updated: June 20, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by 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
Cysts
Digestive System Diseases
Neoplasms
Pathological Conditions, Anatomical

ClinicalTrials.gov processed this record on September 18, 2014