Background 

The STOP-PKD trial is investigating a new treatment option for people with autosomal dominant polycystic kidney disease (ADPKD). 

 
ADPKD is a hereditary condition characterised by the formation of numerous small cysts (fluid-filled sacs) in the kidneys. Over time, these cysts grow, causing the kidneys to gradually lose their function. Those affected often require dialysis or a kidney transplant by middle age. 

 

Currently, there is only one drug (tolvaptan) that can specifically slow the progression of ADPKD. However, tolvaptan leads to a significant increase in urine output (5-8 liters per day) and, consequently, a significant increase in fluid intake. In addition, the drug can potentially cause liver damage, which is why regular laboratory tests are necessary. For these reasons, the drug is not suitable for all patients with ADPKD and is only prescribed in cases of rapid disease progression. Finding new treatment options for ADPKD is therefore particularly important. 

Objective 

This study aims to investigate whether an already approved drug from the group of so-called SGLT2 inhibitors can positively influence the course of ADPKD. 

 
This drug is typically used to treat type 2 diabetes, heart failure, and other forms of chronic kidney disease. Unfortunately, patients with ADPKD were excluded from the studies on chronic kidney disease, so it is currently unclear whether the drug has the same effect in ADPKD. 

 

This study will examine whether taking the drug slows down the progression of kidney disease and improves prognosis for patients with ADPKD. The safety of the drug for patients with ADPKD will also be examined. In the best-case scenario, maintaining kidney function for longer could significantly improve the health and quality of life of patients with ADPKD. 

Procedure 

Participation in the STOP-PKD trial involves the following steps: 

  1. Preliminary examination (screening): 
    First, it is determined whether the patient meets the necessary inclusion criteria. These include certain laboratory values, medical history, and a detailed medical examination. 

  2. Randomization: 
    Participants are randomly assigned to one of two groups: 

  3. Treatment group: receives the SGLT2 inhibitor in tablet form. 

  4. Control group: receives an identical-looking placebo that does not contain any active ingredient. 
    Neither the patients nor the doctors know who is receiving which treatment (this is known as a double-blind procedure). 

  5. Duration of participation: 
    The study will run for approximately three years. During this time, regular clinic visits will take place every three months. Some of these visits can also be carried out close to home by the attending nephrologist. 

  6. Examinations during the study: 

  7. Blood and urine samples will be taken to check kidney function and other health parameters. 

  8. Blood pressure and weight checks, as these play an important role in ADPKD. 

  9. Questionnaires to assess quality of life and disease-specific symptoms. 

  10. Imaging examinations (MRI) for the first 150 patients at the start of the study and after one year. This is done to monitor the growth of the kidneys. 

  11. Safety checks: 
    All side effects and comorbidities are documented. If problems arise, a decision is made on an individual basis as to whether treatment can be continued. 

  12. Voluntary participation: 
    Participation is voluntary. All patients can withdraw their consent at any time without any disadvantages for their further treatment.