Kidney, Polycystic, Autosomal Dominant
Conditions
Keywords
Autosomal Dominant Polycystic Kidney Disease, Polycystic Kidney Disease, Water Loading, Biomarkers
Brief summary
Polycystic kidney disease (PKD) is a genetic disease that occurs in 1 in 500 individuals and leads to kidney failure in half of all affected. Currently, no treatments exist for PKD. PKD-affected kidney cells divide and multiply inappropriately, and form fluid-filled sacs called cysts. Kidney cysts continue to grow throughout life, destroying normal kidney tissue, leading to kidney failure. Based on evidence from basic science research it is believed that drinking high amounts of water can slow the abnormal cysts growth. This study aims to look at changes in urine composition with high water intake in PKD-affected persons compared to healthy individuals.
Interventions
OTHERWater
Participants will be first asked to drink 6 8-oz glasses of water over 2.5 hours on the first day, and then about 12 8-oz glasses of water over the course of the day for one week.
Sponsors
NYU Langone Health
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
Yes
Inclusion criteria
* Clinical diagnosis of Autosomal Dominant Polycystic Kidney Disease by history, ultrasound, CT or MRI * Healthy subjects without a diagnosis of Polycystic Kidney Disease by history, ultrasound, CT or MRI * Ages between 18 and 65 * Healthy subjects (without Polycystic Kidney Disease) must have an estimated glomerular filtration rate (eGFR by the MDRD equation) \> 60 ml/min/1.73 m2 with no history of kidney disease
Exclusion criteria
* Women who are pregnant or nursing * Active dependency on drugs or alcohol * Diagnosis of syndrome of inappropriate antidiuresis * Currently taking a vasopressin agonist or antagonist * Blood sodium level less than \< 135 mEq/L * For healthy participants, estimated glomerular filtration rate (level of kidney function) less than \< 60 ml/min/1.73 m2
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Urine cAMP Concentration and Urine Osmolality (UOsm) | pre- and post-water loading (2 hours) | Urine cAMP (UcAMP) concentration and urine osmolality UOsm) were measured pre- and post-water loading: with 2.5L over 2 hours |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Polycystic Kidney Disease (PKD) Patients | 13 |
| Healthy Patients | 10 |
| Total | 23 |
Baseline characteristics
| Characteristic | Polycystic Kidney Disease (PKD) Patients | Healthy Patients | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 13 Participants | 10 Participants | 23 Participants |
| Sex: Female, Male Female | 8 Participants | 7 Participants | 15 Participants |
| Sex: Female, Male Male | 5 Participants | 3 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 13 | 0 / 10 |
| serious Total, serious adverse events | 0 / 13 | 0 / 10 |
Outcome results
Primary
Change in Urine cAMP Concentration and Urine Osmolality (UOsm)
Urine cAMP (UcAMP) concentration and urine osmolality UOsm) were measured pre- and post-water loading: with 2.5L over 2 hours
Time frame: pre- and post-water loading (2 hours)
Population: Number of participants was deemed to be adequate for the purposes of this pilot study
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Polycystic Kidney Disease Patients | Change in Urine cAMP Concentration and Urine Osmolality (UOsm) | -2.6 fold change in cAMP/UOsm (umol/UOsm) | Standard Error 0.2 |
| Healthy Controls | Change in Urine cAMP Concentration and Urine Osmolality (UOsm) | -2.0 fold change in cAMP/UOsm (umol/UOsm) | Standard Error 0.1 |