A Prospective Observational Study of Foam Sclerotherapy .

NCT ID: NCT04111692

Last Updated: 2024-12-27

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-11-06
• Study Completion Date: 2027-11-30

Brief 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 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.

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 Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Age ≥18 years
• Clinical diagnosis of ADPKD or ADPLD
• 1-3 large, non-exophytic liver or kidney cyst
• Compressive symptoms from dominant cyst

Exclusion Criteria

• 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 100 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Mayo Clinic

OTHER

Sponsor Role: lead

Responsible Party

Marie Hogan

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Marie Hogan, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Mayo Clinic

Rochester, Minnesota, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Cassie Howe, CRC

Role: CONTACT

Phone: 507-266-1230

Email: howe.cassie@mayo.edu

Facility Contacts

Marie C Hogan, MD, PhD

Role: primary

Marie C. Hogan, M.D., Ph.D.

Role: backup

Related Links

https://www.mayo.edu/research/clinical-trials

Mayo Clinic Clinical Trials

Other Identifiers

17-006399

Identifier Type: -

Identifier Source: org_study_id