Polycystic kidney disease causes fluid-filled cysts to grow in the kidneys, gradually replacing healthy tissue. About 1 in 500 people have the inherited form (autosomal dominant PKD), which typically leads to kidney failure in middle age. Treatment has focused on managing blood pressure and symptoms, but tolvaptan became the first drug shown to slow cyst growth.
What's actually going on in research
Trials are testing drugs that target the molecular signals driving cyst formation, including somatostatin analogs and drugs affecting cAMP pathways. Researchers are studying combination approaches, earlier intervention before significant kidney damage occurs, and treatments for the liver cysts and pain that affect many people with PKD. Gene therapy approaches are in early stages.
Cyst growth inhibitors
Several drugs target the cellular signals that make cysts grow. Trials are testing whether somatostatin analogs and other agents can work alongside or instead of tolvaptan, possibly with fewer side effects.
Earlier intervention
Studies are exploring whether starting treatment before kidney function declines can preserve more tissue. This requires better biomarkers to predict who will progress fastest.
Liver and pain management
New trials address liver cysts, which can become massive and painful in PKD. Researchers are also testing treatments for the chronic pain many people experience from kidney size and cyst complications.
What to know before you search
Eligibility typically depends on PKD type (PKD1 or PKD2 gene), kidney size and function, age, blood pressure control, and whether you've tried tolvaptan.
What types of trials are currently open
- Disease progression trials — Testing drugs that slow kidney cyst growth and preserve function, often measured by kidney volume on imaging and decline in filtration rate.
- Kidney function trials — Studies of treatments for people whose PKD has already reduced kidney function significantly, aiming to delay dialysis or transplant.
- Liver cyst trials — Testing drugs or procedures to shrink liver cysts, which can grow large enough to cause pain and limit eating.
- Pain management studies — Trials of treatments for chronic kidney pain from cyst size and complications, which affects quality of life.
- Natural history studies — Following people with PKD over time to understand disease progression patterns and identify who needs treatment most urgently.
Recently added Polycystic Kidney Disease trials
A Pilot Study for Managing Depressive Symptoms in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
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.
Complete imaging scans to evaluate kidney treatment outcomes
Autosomal dominant polycystic kidney disease (ADPKD) is an inherited cystic disorder characterised by the progressive degeneration of the renal parenchyma into cystic formations, with involvement of other organs to varying degrees and incidence (liver, pancreas and brain). This condition is the most common inherited kidney disorder; in fact, it affects 1 in 400-1,000 births and has a prevalence of 5% among dialysis patients and an incidence of 10% among patients with end-stage renal failure in Europe. It is caused by mutations in the PKD1 or PKD2 genes, which are involved in the production of an abnormal protein that leads to tubular dysplasia. Cystic degeneration leads to progressive loss of renal function, with the development of hypertension, haematuria and concomitant enlargement of the renal parenchyma. The progression of the disease is precisely marked by an increase in renal volume. The increase in the organ's overall volume is secondary to the development and enlargement of cysts, whilst the proportion of functioning renal parenchyma progressively decreases. For these reasons, the increase in renal volume over time is a powerful predictor of the risk of end-stage renal disease (ESRD). In addition to its prognostic significance, the enlargement of the kidneys is itself a cause of complications. Indeed, the space occupied within the abdomen can become so extensive as to cause abdominal distension, malaise, pain, loss of appetite, constipation, nausea and vomiting, reduced diaphragmatic movement, breathing difficulties and lower back pain. Overall, patients' quality of life can be severely compromised. It is not uncommon for the kidneys of patients with ADPKD to occupy the pelvic cavity, the preferred site for kidney transplant placement, which represents the optimal treatment option for the disease once ESRD has been reached. This situation, which is not uncommon, represents a temporary contraindication to kidney transplantation: delaying the procedure also has repercussions on the patient's survival. The contraindication to transplantation due to anatomical unavailability has so far necessitated surgical nephrectomy (so-called 'debridement nephrectomy') as the sole preventive or pre-transplant therapeutic option. Nephrectomy carries the risks inherent in surgery, including haemorrhage, herniation of the abdominal wall, vascular complications of varying severity-such as arteriovenous fistulas, thrombosis, and vascular wall injury-and the risk of infection. Surgical nephrectomy also has a negative impact on the subsequent possibility of using the peritoneal membrane for dialysis (peritoneal dialysis) and, should blood transfusions be required to correct intraoperative blood loss, contributes to increasing the likelihood of the patient becoming immunised, with the associated risks of reduced availability of compatible donors (so-called hyperimmune patients), and, in any case, a higher risk of acute and chronic rejection, conditions that negatively impact transplant survival. Given the high risks associated with nephrectomy, a non-invasive alternative has been proposed: reduction of renal volume via transcatheter arterial embolisation. Renal embolisation can be performed in the Interventional Radiology department via the controlled occlusion of renal vessels using a liquid embolisation agent composed of ethylene vinyl alcohol (EVOH). The literature reports the assessment of embolised patients using CT without contrast medium, but recent technological innovations allow for accurate and precise volumetric assessment of organs using MRI without contrast medium, with reduced inter-operator variability and without the need to subject the patient to ionising radiation during follow-up.
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