Clinical Studies

For drugs and therapies to be developed for PKD, clinical studies must be conducted. Patients play a key part. Learn how you can help unlock the secrets of PKD and find a treatment by participating in a study.


Participate in PKD Clinical Studies to Discover Treatments and Improve Your Health


Types of Clinical Studies

The two types of studies currently underway for PKD patients include observational studies and clinical trials.

  • Observational studies monitor and record various clinical and/or subjective factors over a period oftime, like blood pressure or quality of life, to better understand how the disease progresses. Involvement includes activities such as having conversations with researchers and physicians, or recording daily events in journals. No drugs are given or other interventions are made to alter the course of the disease. An example of an observational study is the Consortiumfor Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP).
  • Clinical trials monitor and record various clinical and/or subjective parameters by testing experimental treatments, devices or combinations of drugs that may alter the course of the disease. Participants receive specific interventions which may be medical products such as drugs or devices; procedures; orchanges to participants’ behavior such as diet. The purpose of a clinical trial is to test a new drug or other intervention for safety and effectiveness in treating the disease in question before it can be prescribed for patients. An example of a clinical trial is theTolvaptan TEMPO ¾ Trial.

According to, an intervention is a processor action that is the focus of a clinical study. This can include giving participants drugs, medical devices, procedures,vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches such as surveys, education and interviews.

Phases of a Clinical Trial

A new drug to treat PKD must move through each stage before it can be reviewed for approval by the FDA.

  • Phase I – The new drug is tested for safety and side effects in a small number of healthy volunteers withPKD.
  • Phase II – The new drug is tested for safety, doseranging and preliminary effectiveness in a small number of volunteers.
  • Phase III – The new drug is tested in a large number of volunteers with PKD to establish effectiveness, monitor side effects and compare results with currenttreatments. The data collected during the clinical trial is analyzed and then submitted to the FDA for regulatory review, which can take one to three years. If there is an intent to apply for an NDA (new drug application), it is communicated to the FDA and shapes the design of the study. Once it is approved, the new drug can be prescribed by physicians to treat PKD.
  • Phase IV – These studies are done after the drug has been approved by the FDA and it is in use (considered a post-marketing study).

Clinical Trials Awareness Program (CTAP)

Perhaps the single greatest impediment to clinical trial patient participation/recruitment begins with awareness. We believe it is our goal to address this challenge and launched the Clinical Trials Awareness Program, an aggressive program to help create awareness amongst patients and families and speed up clinical trial recruitment. The program is focused on educating patients about current studies so they can make educated decisions about participating. The goal is to simplify the process of finding clinical studies for PKD patients in their geographic area by sending Accelerating Clinical Trials (ACT) Alert emails about studies that are being conducted.

Sign Up to Receive ACT Alerts

Learn more about the Clinical Trials Awareness Program

Participate in a Clinical Trial

To participate in a clinical trial, you will need to have a formal diagnosis of PKD made by a physician. Learn more about the implications of a PKD diagnosis for yourself or your children.

By participating in a clinical trial, you can:

  • Play a more active role in your own health care.
  • Have access to new treatments before they are available to the public.
  • Contribute to the development of treatments for PKD.
  • Make things better for the next generation of those with PKD.

Your Organization

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Phone: 800.443.9441 | Fax: 843.216.6100

©2014, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.

©2014, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.