Autosomal dominant polycystic kidney disease (ADPKD) is an inherited disease that leads to chronic kidney disease (CKD). If a person has ADPKD, there is a 50 percent chance their children may also develop ADPKD.

People with ADPKD have multiple fluid-filled sacs, called cysts, which grow and multiply on their kidneys. The cysts cause serious damage to the kidneys and interfere with normal kidney function. Over time, this damage may lead to kidney failure.

These sites provide more information about ADPKD and other chronic kidney diseases (available in English only):

PKD Foundation
National Organization for Rare Disorders

About Clinical Research

Clinical research studies (also called clinical trials) are done to test investigational treatments for diseases and conditions. Studies determine if treatments are safe to use and work to improve the health of people. Before a medication, therapy, or medical device can be made available to the general public, it has to go through several phases of clinical research. All prescription medications that are sold and used today have been tested in clinical studies.

Why do people participate in studies?

Clinical study participation is completely voluntary. People participate in clinical research studies for a variety of reasons. Some volunteer to participate in studies because they want to learn more about their disease. Others volunteer because they want to help researchers learn more about a disease so that it may help others in the future. Although a participant can always change his or her mind, completion of the study by all participants is important. Please carefully consider your ability to participate fully.

Are clinical studies safe?

Clinical research studies follow a specific set of standards and are closely regulated to ensure the safety of all participants. Safety precautions are put in place to protect people who participate in clinical research. In addition, studies follow a written plan that is called a protocol. The protocol is designed to make sure all study procedures are conducted correctly and safely.

What if I have questions during the study?

You can ask questions of the study team at any point before, during, and after the study. Before agreeing to participate, please make sure that you understand the responsibilities of study participants. If you have any concerns about participating in the study, you should feel comfortable discussing them with a member of the study team at any time.

Can I leave the study once I have agreed to participate?

Study participation is completely voluntary, and you can end your participation at any time, for any reason. If you think you would like to stop participating in the study, talk to the study doctor or study staff. If you decide to leave the study early, the study doctor will ask that you return to the study site for a final visit, but there will be no negative impact on the care you receive.

Can I see other doctors during the study?

During the study, you can still see your regular doctor for non-study-related care. You should tell the study doctor if you start taking any other medications.

About the Study

What is the purpose of the study?

The purpose of the REPRISE study is to evaluate the safety and effectiveness of an investigational medication called tolvaptan in patients with late stage 2 to early stage 4 chronic kidney disease caused by autosomal dominant polycystic kidney disease (ADPKD). The kidney function of participants will be checked through frequent blood tests.

What is the investigational study medication?

The investigational medication being studied is tolvaptan.

The kidneys of people who have ADPKD do not respond correctly to a hormone called vasopressin. It is believed that vasopressin plays a role in the development and growth of kidney cysts. It is hoped that tolvaptan will interfere with or block the effect that vasopressin has on the kidneys to reduce cyst growth and delay kidney failure.

You will take the study medication twice a day. You should take the first dose of study medication when you wake up and the second dose approximately 9 hours later.

How long will the study last?

If you are eligible for the study, your participation will last approximately 15 months. Once you have completed the REPRISE study, you may be able to participate in an extension study.

Who can participate?

You may be able to join the study if you are:

  • Between the ages of 18 to 55 years old; patients aged 56 to 65 may be able to participate in some circumstances
  • Diagnosed with ADPKD

There are other criteria that you must meet to participate. The study staff can discuss these criteria with you in greater detail.

What will happen during the study?

If you are eligible and agree to participate in the study, you will be required to:

  • Visit the study clinic up to 25 times. Depending on your circumstances, you may be offered an opportunity to have certain study visits performed at your home or another location more convenient than the study clinic, if this option is available in your region
  • Take the investigational medication twice a day as directed by the study doctor
  • Have blood and urine tests
  • Drink enough fluids (a minimum of 8 to 12 glasses during the day)