Polycystic kidney disease causes fluid-filled cysts to grow in the kidneys, eventually damaging tissue and reducing function. About 500,000 people in the U.S. have PKD, most with the autosomal dominant form inherited from one parent. Until recently, treatment focused on managing blood pressure and complications, but newer drugs can now slow cyst growth and preserve kidney function longer.
What's actually going on in research
Trials are testing drugs that slow cyst growth through different pathways, including vasopressin receptor blockers like tolvaptan (already approved), SGLT2 inhibitors borrowed from diabetes care, and therapies targeting the cellular signals that drive cyst formation. Researchers are also studying biomarkers to predict who will progress fastest and when to start treatment.
SGLT2 inhibitors
Diabetes drugs like dapagliflozin are being tested in PKD after showing they protect kidneys in other conditions. Early results suggest they may slow kidney function decline in people with PKD.
Early intervention
Studies are testing whether starting tolvaptan earlier in disease course, before significant function loss, prevents more kidney damage. Researchers are identifying imaging and genetic markers to predict who needs earlier treatment.
Cyst growth pathways
Several experimental drugs target the molecular signals that tell kidney cells to form cysts. These include therapies aimed at cAMP, mTOR, and other pathways active in PKD.
What to know before you search
Eligibility typically depends on kidney function level, kidney size or growth rate on imaging, PKD genotype, and whether you've taken tolvaptan or other PKD treatments before.
What types of trials are currently open
- Disease-slowing trials — Testing drugs that aim to slow cyst growth and preserve kidney function, measured by kidney volume on MRI and filtration rate over time.
- Repurposing trials — Testing drugs approved for other conditions, like diabetes or heart failure medications, to see if they slow PKD progression.
- Biomarker studies — Following people with PKD to identify blood tests, urine markers, or imaging features that predict how fast the disease will progress.
- Symptom management trials — Testing treatments for PKD complications like pain from cyst bleeding or rupture, high blood pressure, and kidney stones.
- Genetic studies — Studies examining how specific PKD1 or PKD2 gene mutations affect disease severity and treatment response.
Recently added Polycystic Kidney Disease trials
Take an experimental oral medicine at increasing doses
This is a first-in-human, multi-part clinical study designed to evaluate the safety, tolerability, pharmacokinetics (PK), food effect, and drug-drug interaction (DDI) potential of GV-100 following oral administration in healthy participants. The study is divided into four parts: Single Ascending Dose (SAD), Multiple Ascending Dose (MAD), Food Effect (FE), and Drug-Drug Interaction (DDI).
Try digital therapy or coaching to manage depression and pain
The purpose of this research is to evaluate the feasibility and effectiveness of Digital Neurotherapy (DNT) or Capacity Coaching (CC) for managing symptoms of depression and self reported pain in Autosomal Dominant Polycystic Kidney Disease (ADPKD) compared to education alone.
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