Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT02173080 |
| Other study ID # |
14-003832 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
June 2014 |
| Est. completion date |
July 2024 |
Study information
| Verified date |
November 2023 |
| Source |
Mayo Clinic |
| Contact |
Marie C Hogan, MD, PhD |
| Phone |
507-266-1963 |
| Email |
Hogan.marie[@]mayo.edu |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
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).
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.
