Polycystic kidney disease causes fluid-filled cysts to grow in the kidneys, gradually replacing normal tissue. About 1 in 500 people have the inherited form, making it one of the most common genetic diseases. Most people eventually develop kidney failure and need dialysis or transplant, though the timeline varies widely.
What's actually going on in research
Trials are testing drugs that slow cyst growth, including vasopressin receptor antagonists like tolvaptan (FDA-approved in 2018), SGLT2 inhibitors borrowed from diabetes care, and mTOR inhibitors. Researchers are also studying drugs that target the proteins made by the mutated PKD genes, pain management approaches, and whether slowing cyst growth earlier in life improves long-term outcomes.
SGLT2 inhibitors
Diabetes drugs like dapagliflozin and empagliflozin appear to protect kidney function in PKD. Trials are testing whether they can slow disease progression with fewer side effects than tolvaptan.
Earlier intervention
Studies are testing whether starting treatment before significant kidney damage occurs prevents later decline. This shifts focus from waiting until kidneys are failing to treating based on cyst burden and growth rate.
Pain management
Chronic pain affects many people with PKD but treatment options are limited. Trials are testing nerve blocks, medications, and procedures to drain or remove large cysts causing discomfort.
What to know before you search
Eligibility typically depends on genetic confirmation of PKD, kidney size or growth rate on imaging, current kidney function level, blood pressure control, and whether you've tried tolvaptan.
What types of trials are currently open
- Disease-slowing trials — Testing drugs that aim to slow cyst growth and preserve kidney function, often measured by kidney size on imaging and blood creatinine levels over one to three years.
- Kidney protection trials — Studies of medications already approved for other conditions, like blood pressure drugs or diabetes medications, to see if they help PKD kidneys last longer.
- Pain management trials — Testing treatments for chronic kidney or abdominal pain, including medications, nerve procedures, and cyst drainage techniques.
- Genetic modifier studies — Research looking at other genes that might explain why PKD progresses faster in some families than others.
- Quality of life studies — Following people with PKD to understand how symptoms like pain, fatigue, and anxiety affect daily life and what helps.
Recently added Polycystic Kidney Disease trials
Take a single dose of an experimental oral medication
The purpose of the study is to evaluate the pharmacokinetics, safety and tolerability of VX-407 following a single oral dose.
Take a new drug alongside your birth control pill
The purpose of the study is to evaluate the pharmacokinetic effects and safety and tolerability of VX-407 when coadministered with oral contraceptives.
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