Renal

Autosomal Dominant Polycystic Kidney Disease

Also known as ADPKD, PKD1/PKD2 disease, adult polycystic kidney

Autosomal dominant polycystic kidney disease is the most common inherited kidney disorder, caused by mutations in PKD1 or PKD2 encoding polycystin-1 and polycystin-2 respectively, leading to progressive bilateral renal cyst development and

ORPHA:730 ↗Gene PKD1Gene PKD2Prevalence Approximately 1 in 400–1,000; one of the most common monogenic disordersOnset Adult (symptoms typically in 3rd–4th decade; cysts present from birth)

31

studies recruiting now

as of 7 Sept 2026

197

studies registered in total

as of 7 Sept 2026

14

countries with a recruiting site

as of 7 Sept 2026

26 Aug 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 31 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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Support

Patient organisations

PKD FoundationPatient association
Visit website ↗

Registry: ADPKD Registry (CRISP Study / HALT-PKD Data) · Join ↗. Registries connect patients to researchers and often hear about trials first.

About Autosomal Dominant Polycystic Kidney Disease

Autosomal dominant polycystic kidney disease is the most common inherited kidney disorder, caused by mutations in PKD1 or PKD2 encoding polycystin-1 and polycystin-2 respectively, leading to progressive bilateral renal cyst development and enlargement. It is the fourth leading cause of end-stage renal disease globally, with extrarenal manifestations including intracranial aneurysms, hepatic cysts, and mitral valve prolapse. PKD1 mutations cause a more severe phenotype than PKD2, with earlier onset of renal failure.

Common clinical features

Bilateral enlarged kidneys with multiple cystsHypertension (often the earliest manifestation)Flank or abdominal painHaematuriaUrinary tract infections and cyst infectionsProgressive decline in eGFRIntracranial aneurysms and hepatic cysts (extrarenal)

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

1 approved treatment and 24 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Tolvaptan (Jinarc)
Phase 3Somatostatin
Phase 3Pravastatin
Phase 3Metformin
Phase 3Hydrochlorothiazide (Apo-hydro)
Phase 3Spironolactone (Abbolactone)
Phase 3Everolimus (Afinitor)
Phase 3Bardoxolone Methyl
Phase 3Octreotide

+ 16 more in development

Before you apply

Things trial teams commonly ask about for Autosomal Dominant Polycystic Kidney Disease. Not eligibility rules; those are set by each study.

  • Total kidney volume (TKV) measured by MRI or CT is the primary imaging biomarker and is used for Mayo Clinic Classification (class 1A–1E); document TKV and height-adjusted TKV before applying.
  • Tolvaptan eligibility criteria typically require rapidly progressive disease (Mayo class 1C–1E or historical TKV growth >5% per year); gather longitudinal imaging if available.
  • Current eGFR and rate of decline over the preceding 1–3 years are key eligibility metrics; ensure creatinine history is compiled from your GP or nephrologist.

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).