Polycystic Liver DiseaseNCT02173080
Development and Assessment of The Polycystic Liver Disease Questionnaire (PLD-Q).
An observational study of Polycystic Liver Disease, run by Mayo Clinic at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycystic Liver Disease
- What is being tested?
- PLD-Q
- How many people
- 594 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2014to 11 Mar 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with polycystic liver disease
Who may be able to join
- Patients ≥ 18 year
- Polycystic liver, defined by > 20 liver cysts on imaging
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Development and Assessment of the Psychometric Properties of a Polycystic Liver Disease-specific Patient Reported Outcomes Questionnaire (PLD-Q).
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).
Read the 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.
- Conditions
- Polycystic Liver Disease
- Being tested
- PLD-Q
- Keywords
- autosomal dominant polycystic liver disease, autosomal dominant polycystic kidney disease, quality of life, patient reported outcome tool
- Medical subject headings
- Polycystic liver disease, Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PLD-QOther
polycystic liver disease questionnaire
| Study group | Role | What they receive |
|---|---|---|
| Polycystic liver disease patients | will receive PLD-Q, EORTC QLQ-30 symptoms subscale, EQ5D-VAS score and SF36 Receives: Other: PLD-Q | |
| ADPKD group without PLD | will receive PLD-Q | |
| Healthy controls | receive PLD-Q Receives: Other: PLD-Q | |
| PLD patient focus group | to discuss and improve PLD-Q | |
| PLD clinical expert focus group | to discuss and improve PLD-Q |
What is being measured
The primary outcome is the main question the study is designed to answer.
Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q) total score
total score on the PLD-Q
Measured: baseline to 6 months
Secondary outcomes (5)
The Short Form (36) Health Survey (SF36)
Measured: baseline to 6 months
European Organisation for Research and Treatment of Cancer Quality of life questionnaire (EORTC-QLQ-C30) symptom subscale
Measured: baseline to 6 months
Euroqol group 5 dimensions questionnaire visual analog score (EQ5D-VAS)
Measured: baseline to 6 months
Total liver volume
Measured: baseline
Total kidney volume
measured in patients with autosomal dominant polycystic kidney disease (ADPKD)
Measured: baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2014 (estimated)
- Study startJun 2014
- Primary completion11 Mar 2024
- Study completion11 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycystic Liver DiseaseStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 594 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Marie Hogan, Mayo Clinic
- Investigators
- Marie C Hogan, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02173080
- Sponsor's protocol ID
- 14-003832
- Status last verified
- Sep 2024
- First submitted
- 20 Jun 2014
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development and Assessment of the Psychometric Properties of a Polycystic Liver Disease-specific Patient Reported Outcomes Questionnaire (PLD-Q).
- ClinicalTrials.gov ID
- NCT02173080
- Acronym / sponsor ID
- 14-003832
- Conditions
- Polycystic Liver Disease
- Keywords
- autosomal dominant polycystic liver disease, autosomal dominant polycystic kidney disease, quality of life, patient reported outcome tool
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 594 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Jun 2014 (actual)
- Primary completion
- 11 Mar 2024 (actual)
- Study completion
- 11 Mar 2024 (actual)
- First posted
- 24 Jun 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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 design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Polycystic liver disease patients | – | Other: PLD-Qwill receive PLD-Q, EORTC QLQ-30 symptoms subscale, EQ5D-VAS score and SF36 |
| ADPKD group without PLD | – | –will receive PLD-Q |
| Healthy controls | – | Other: PLD-Qreceive PLD-Q |
| PLD patient focus group | – | –to discuss and improve PLD-Q |
| PLD clinical expert focus group | – | –to discuss and improve PLD-Q |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with polycystic liver disease
Key inclusion criteria (2 listed)
- Patients ≥ 18 year
- Polycystic liver, defined by > 20 liver cysts on imaging
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PLD-Qpolycystic liver disease questionnaire | Other | Healthy controls, Polycystic liver disease patients |
Primary outcome
Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q) total score
Time frame: baseline to 6 months
total score on the PLD-Q
5 secondary outcomes
The Short Form (36) Health Survey (SF36)
Time frame: baseline to 6 months
European Organisation for Research and Treatment of Cancer Quality of life questionnaire (EORTC-QLQ-C30) symptom subscale
Time frame: baseline to 6 months
Euroqol group 5 dimensions questionnaire visual analog score (EQ5D-VAS)
Time frame: baseline to 6 months
Total liver volume
Time frame: baseline
Total kidney volume
Time frame: baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
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Polycystic Liver Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Marie C Hogan principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Polycystic Liver Disease, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Polycystic Liver Disease-specific patient reported outcomes questionnaire (PLD-Q) total score (time frame: baseline to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.