A Clinical Trial of Water Therapy for Autosomal Dominant Polycystic Kidney Disease

Patients affected by Autosomal Dominant Polycystic Kidney Disease (ADPKD) need a safe and effective long-term treatment regimen. Unfortunately, there are still no disease-specific treatment for ADPKD approved in the US. A rational step towards identifying such agents is to test therapies that have a proven safety profile with mechanisms of action that can counter the disease progression.

The purpose of this study is to investigate whether drinking increased amounts of water (water loading) might slow down polycystic kidney growth or kidney function decline. Water loading can cause the suppression of a pathway that causes fluid buildup and cyst growth. High water intake has been safely used in the clinical setting, such as in the case of kidney stone therapy. New York State tap water is widely available and safe, making it highly cost-effective as well.

Study Overview

• Status: Completed
• Conditions: Autosomal Dominant Polycystic Kidney
• Intervention / Treatment: Other: High Water Intake

Detailed Description

The study will involve 11 visits to the study site over 19 months. Participants will need to follow specific dietary and fluid recommendations. There will be physical examinations and medical history assessments at each visit. Testing will include undergoing magnetic resonance imaging (MRI), blood and urine tests. Study participants will be compensated for their time. Detailed study procedures will be reviewed upon contact with the study team.

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10021
      • The Rogosin Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• pre-existing diagnosis of Autosomal Dominant Polycystic Kidney Disease
• estimated glomerular filtration rate of 40 ml/min or greater
• urine osmolality > 400 mOsm/L

Exclusion Criteria:

• estimated glomerular filtration rate less than 40 ml/min
• low blood sodium levels
• syndrome of inappropriate diuretic hormone
• use of thiazide diuretics or selective serotonin reuptake inhibitors (SSRIs)
• use of tolvaptan, another vasopressin receptor antagonist, vasopressin agonists or dDAVP
• contraindications to magnetic resonance imaging (MRI) (pacemakers, defibrillators, implanted electronic devices, metallic foreign body)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
No Intervention: Usual Water Intake; For the first 6 months of the study, the participants will continue their usual water intake.
Experimental: High Water Intake; After a 6 month period of usual water intake, a high water intake daily amount will be prescribed for 1 year.	Other: High Water Intake; After 6 months of usual, unchanged diet and fluid intake, participants will be asked to increase the daily fluid intake based on the principal investigator's prescription. The actual amount of extra water prescribed will depend on the results of the participant's 24 hour urine test.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in total kidney volume, as measured from magnetic resonance imaging	Total kidney volumes will be measured before and after the period of high water intake. Kidney volume growth with high water intake will be compared to baseline kidney volume growth.	18 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Kidney function change	Blood creatinine levels will be measured and compared before and after the high water intake period.	18 months
Change in urine and blood markers of response to high water intake	Blood and urine biomarkers of response to high water intake will be measured before and after the period of high water intake.	18 months.

Collaborators and Investigators

• Sponsor: The Rogosin Institute
• Collaborators: Weill Medical College of Cornell University
• Investigators: Principal Investigator: Martin Prince, MD, Weill Medical College of Cornell University

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2017
• Primary Completion (Actual): December 31, 2021
• Study Completion (Actual): December 31, 2021

Study Registration Dates

• First Submitted: March 27, 2017
• First Submitted That Met QC Criteria: March 30, 2017
• First Posted (Actual): April 6, 2017

Study Record Updates

• Last Update Posted (Actual): October 19, 2023
• Last Update Submitted That Met QC Criteria: October 17, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Joint Diseases; Musculoskeletal Diseases; Muscular Diseases; Musculoskeletal Abnormalities; Abnormalities, Multiple; Kidney Diseases, Cystic; Ciliopathies; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant; Arthrogryposis
• Other Study ID Numbers: 1701017921

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No