Autosomal Dominant Polycystic KidneyNCT04111692
A Prospective Observational Study of Foam Sclerotherapy .
An observational study of Autosomal Dominant Polycystic Kidney and Renal Cyst, run by Mayo Clinic at 1 site in United States.
Trial summary
An observational prospective study to determine the impact of foam sclerotherapy of large, dominant kidney/liver cysts on quality of life outcomes and kidney/liver cyst volumes at up to 12 months of follow-up in patients with autosomal dominant polycystic kidney disease (ADPKD) and autosomal dominant polycystic liver… Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney, Renal Cyst, Autosomal Dominant Polycystic Liver Disease
- How many people
- 100 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Nov 2017to Nov 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult subjects with ADPKD or ADPLD and large, symptomatic kidney or liver cysts followed in Nephrology or Hepatology clinics at Mayo Clinic, or de novo referrals to Mayo Clinic, Rochester, MN will be invited to participate.
Who may be able to join
- Age ≥18 years
- Clinical diagnosis of ADPKD or ADPLD
- 1-3 large, non-exophytic liver or kidney cyst
- Compressive symptoms from dominant cyst
Who cannot join
- Age <18 years
- Absence of ADPKD or ADPLD
- Largest cyst <4cm in all 3 dimensions or largest cyst volume <400cc
- >3 large, non-exophytic liver or kidney cyst(s) ≥4cm in all 3 dimensions (cyst volume ≥400cc)
- Unwilling to comply with study follow-up protocol to 12 ± 1months post-FS
- Lacking capacity to provide informed consent to FS of liver or kidney cysts
- Pregnancy
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Marie C Hogan, MD, PhD, 507-266-1963, hogan.marie@mayo.edu
Marie C. Hogan, M.D., Ph.D.
About this study
Official title: A Prospective Observational Study of Foam Sclerotherapy for the Treatment of Symptomatic Kidney or Liver Cysts in Patients With Autosomal Dominant Polycystic Kidney and Liver Disease.
An observational prospective study to determine the impact of foam sclerotherapy of large, dominant kidney/liver cysts on quality of life outcomes and kidney/liver cyst volumes at up to 12 months of follow-up in patients with autosomal dominant polycystic kidney disease (ADPKD) and autosomal dominant polycystic liver disease (ADPLD).
Read the detailed description
Cyst burden is an important determinant of outcomes in both autosomal dominant polycystic kidney disease (ADPKD) (1, 2) and autosomal dominant polycystic liver disease (ADPLD) (3, 4). Furthermore, mass symptoms (from liver and kidney volume) greatly impact upon quality of life in patients with severe disease. Cyst volume increases exponentially with age and results in the development of end-stage renal disease and hypertension, compromised quality of life due to compressive symptoms, and predisposes patients to cyst complications such as infection, hemorrhage, rupture, and torsion. Existing percutaneous treatments for cyst burden in ADPKD and ADPLD include cyst aspiration with or without sclerotherapy. Although frequently effective in the short-term, recurrence rates and the need for repeat procedures are high after these procedures (5, 6). Extrarenal disease (primarily liver disease) is the most important aspect of disease burden to ADPKD patients (7), and there are few effective treatments.
Foam sclerotherapy (FS) with 3% Sodium Tetradecyl Sulfate (STS) a sclerosing agent. (Sotradecol®; Mylan, Galway, Ireland) is approved by the FDA for the management of varicose veins. While increasing cyst burden significantly compromises quality of life, the impact of FS on patient-reported outcomes has not been evaluated. In collaboration with the Center for Science of Healthcare delivery, we have developed a patient-reported outcome tool for polycystic liver disease capable of detecting symptom burden in individuals with polycystic liver disease that has been approved by the FDA as a patient-reported outcomes tool in research. Furthermore measurement of liver and kidney volumes can be performed in the Polycystic Kidney Disease Imaging Research Core that monitors organ volumes before and after interventions. At this time, patients are interested in procedures that will alleviate and palliate their mass symptoms but desire preliminary information on the procedural efficacy. We aim to report our experience with a new therapeutic advance - FS for the treatment of liver and kidney cysts at Mayo Clinic - and to determine the impact of this procedure on patient-reported quality of life measures and changes in organ volumes.
- Conditions
- Autosomal Dominant Polycystic Kidney, Renal Cyst, Autosomal Dominant Polycystic Liver Disease, Hepatic Cyst
- Keywords
- hepatic cyst, Kidney Cyst, Autosomal Polycystic Liver Disease, Autosomal Polycystic Kidney Disease
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Pain Inventory
Measured using self assessed brief pain inventory (short form) questionnaire, using a scale of 0 is no pain and 10 is pain as bad as you can imagine
Measured: Baseline, 1 month, 6 month, 12 month
Change in Self Assessed Quality of life
Measured using the linear analogue self assessed questionnaire, using a scale of 0 is as bad as it can be and 10 is as good as it can be
Measured: Baseline, 1 month, 6 month, 12 month
Change in health related quality of life
Measured using the self assessed Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36), using a scale that ranges from yes and no or all of the time to none of the time
Measured: Baseline, 1 month, 6 month, 12 month
Change in Polycystic Liver Disease Quality of Life
Measured using the self assessed Polycystic Liver Disease Questionnaire, using a scale that ranges from never/not at all to always/a lot
Measured: Baseline, 1 month, 6 month, 12 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2019
- Study start6 Nov 2017
- Primary completionNov 2027 (estimated)
- Study completionNov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autosomal Dominant Polycystic KidneyStudies 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Marie Hogan, Mayo Clinic
- Investigators
- Marie Hogan, MD, PhD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04111692
- Sponsor's protocol ID
- 17-006399
- Status last verified
- Jan 2026
- First submitted
- 1 May 2019
- Last updated
- 26 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Observational Study of Foam Sclerotherapy for the Treatment of Symptomatic Kidney or Liver Cysts in Patients With Autosomal Dominant Polycystic Kidney and Liver Disease.
- ClinicalTrials.gov ID
- NCT04111692
- Acronym / sponsor ID
- 17-006399
- Conditions
- Autosomal Dominant Polycystic Kidney, Renal Cyst, Autosomal Dominant Polycystic Liver Disease, Hepatic Cyst
- Keywords
- hepatic cyst, Kidney Cyst, Autosomal Polycystic Liver Disease, Autosomal Polycystic Kidney Disease
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 6 Nov 2017 (actual)
- Primary completion
- Nov 2027 (estimated)
- Study completion
- Nov 2027 (estimated)
- First posted
- 1 Oct 2019 (actual)
- 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.
An observational prospective study to determine the impact of foam sclerotherapy of large, dominant kidney/liver cysts on quality of life outcomes and kidney/liver cyst volumes at up to 12 months of follow-up in patients with autosomal dominant polycystic kidney disease (ADPKD) and autosomal dominant polycystic liver disease (ADPLD).
Detailed description
Cyst burden is an important determinant of outcomes in both autosomal dominant polycystic kidney disease (ADPKD) (1, 2) and autosomal dominant polycystic liver disease (ADPLD) (3, 4). Furthermore, mass symptoms (from liver and kidney volume) greatly impact upon quality of life in patients with severe disease. Cyst volume increases exponentially with age and results in the development of end-stage renal disease and hypertension, compromised quality of life due to compressive symptoms, and predisposes patients to cyst complications such as infection, hemorrhage, rupture, and torsion. Existing percutaneous treatments for cyst burden in ADPKD and ADPLD include cyst aspiration with or without sclerotherapy. Although frequently effective in the short-term, recurrence rates and the need for repeat procedures are high after these procedures (5, 6). Extrarenal disease (primarily liver disease) is the most important aspect of disease burden to ADPKD patients (7), and there are few effective treatments.
Foam sclerotherapy (FS) with 3% Sodium Tetradecyl Sulfate (STS) a sclerosing agent. (Sotradecol®; Mylan, Galway, Ireland) is approved by the FDA for the management of varicose veins. While increasing cyst burden significantly compromises quality of life, the impact of FS on patient-reported outcomes has not been evaluated. In collaboration with the Center for Science of Healthcare delivery, we have developed a patient-reported outcome tool for polycystic liver disease capable of detecting symptom burden in individuals with polycystic liver disease that has been approved by the FDA as a patient-reported outcomes tool in research. Furthermore measurement of liver and kidney volumes can be performed in the Polycystic Kidney Disease Imaging Research Core that monitors organ volumes before and after interventions. At this time, patients are interested in procedures that will alleviate and palliate their mass symptoms but desire preliminary information on the procedural efficacy. We aim to report our experience with a new therapeutic advance - FS for the treatment of liver and kidney cysts at Mayo Clinic - and to determine the impact of this procedure on patient-reported quality of life measures and changes in organ volumes.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult subjects with ADPKD or ADPLD and large, symptomatic kidney or liver cysts followed in Nephrology or Hepatology clinics at Mayo Clinic, or de novo referrals to Mayo Clinic, Rochester, MN will be invited to participate.
Key inclusion criteria (4 listed)
- Age ≥18 years
- Clinical diagnosis of ADPKD or ADPLD
- 1-3 large, non-exophytic liver or kidney cyst
- Compressive symptoms from dominant cyst
Key exclusion criteria (7 listed)
- Age <18 years
- Absence of ADPKD or ADPLD
- Largest cyst <4cm in all 3 dimensions or largest cyst volume <400cc
- >3 large, non-exophytic liver or kidney cyst(s) ≥4cm in all 3 dimensions (cyst volume ≥400cc)
- Unwilling to comply with study follow-up protocol to 12 ± 1months post-FS
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in Pain Inventory
Time frame: Baseline, 1 month, 6 month, 12 month
Measured using self assessed brief pain inventory (short form) questionnaire, using a scale of 0 is no pain and 10 is pain as bad as you can imagine
Change in Self Assessed Quality of life
Time frame: Baseline, 1 month, 6 month, 12 month
Measured using the linear analogue self assessed questionnaire, using a scale of 0 is as bad as it can be and 10 is as good as it can be
Change in health related quality of life
Time frame: Baseline, 1 month, 6 month, 12 month
Measured using the self assessed Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36), using a scale that ranges from yes and no or all of the time to none of the time
Change in Polycystic Liver Disease Quality of Life
Time frame: Baseline, 1 month, 6 month, 12 month
Measured using the self assessed Polycystic Liver Disease Questionnaire, using a scale that ranges from never/not at all to always/a lot
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2026.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Marie Hogan, MD, PhD 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 Autosomal Dominant Polycystic Kidney and Renal Cyst, 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 4 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 26 Jan 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Cassie Howe, CRC (507-266-1230, howe.cassie@mayo.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Pain Inventory (time frame: Baseline, 1 month, 6 month, 12 month). The study lists 4 primary outcomes in all. 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 26 Jan 2026.