Development & Assessment of The Polycystic Liver Disease Questionnaire

Status Recruiting

Clinical Trial 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).

Clinical Trial 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 Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02173080
Study type	Observational
Source	Mayo Clinic
Contact	Marie C Hogan, MD, PhD
Phone	507-266-1963
Email	Hogan.marie@mayo.edu
Status	Recruiting
Phase
Start date	June 2014
Completion date	July 2024

View Details

See also
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Completed	`NCT00426153` - Octreotide in Severe Polycystic Liver Disease	Phase 2/Phase 3
Terminated	`NCT00934791` - Polycystic Liver Disease in Kidney Transplant	N/A
Completed	`NCT02021110` - Ursodeoxycholic Acid as Treatment for Polycystic Liver Disease	Phase 2
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Active, not recruiting	`NCT00771888` - Open-Label Extension of LOCKCYST Trial	Phase 2/Phase 3
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Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms