Clinical Trials

Without clinical studies, development for new PKD treatments and therapies would be impossible. Patients like you play a key role in the research and development process by volunteering to participate.

Unfortunately, many clinical trials fail because they aren’t able to recruit enough volunteers. This is especially true of PKD treatment development.

By participating in a clinical trial, you can help researchers discover new treatments for PKD, gain access to care from medical experts, and potentially benefit from a medical breakthrough before it’s released to the public.

The U.S. Food and Drug Administration (FDA) defines the four phases of clinical trials. A new drug must pass through each phase before the FDA will review it for approval. Here’s what that looks like for the PKD community:

• Phase I: A small number of healthy volunteers with PKD test a new drug for safety and side effects.
• Phase II: A small number of volunteers test the new drug for safety, dosage, and preliminary effectiveness.
• Phase III: A large number of volunteers with PKD test the new drug. This phase aims to establish effectiveness, monitor side effects, and compare results with current treatments.
• Phase IV: This phase occurs after the FDA approves a new drug. Researchers collect and analyze data about the risks, benefits, and optimal use of the drug.

Before taking part in a clinical trial, you must meet their eligibility criteria and then move to informed consent, a process where you and the study doctor will discuss what participating in the study will be like.

Eligibility Criteria

Every study has different guidelines for who can or cannot participate. These guidelines, called eligibility criteria, describe characteristics that must be shared by all participants. The criteria differs from study to study.

For PKD studies, eligibility criteria may include age, gender, medical history, and current kidney function. Eligibility criteria for PKD treatment studies often require that patients have a particular risk of progression or stage of PKD.

Specific eligibility criteria help control for certain variables so that researchers can achieve accurate and meaningful results. It also helps protect vulnerable individuals from increased risks.

Eligibility criteria are approved by ethics review boards, and no exceptions can be made.

Informed Consent

The informed consent form will outline the purpose of the research, its duration, and a list of the procedures you will undergo.

The form should also address participation risks, how your privacy will be protected, and contact information for the study team.

Remember, by signing an informed consent form, you are not signing a contract. You can leave the study at any time, and the people who monitor the trial can also remove you if they feel your health is declining.

Time Commitment

Time is one of the biggest commitments you’ll make when you participate in a clinical trial.

It is likely you’ll need to attend frequent checkups and undergo tests, like blood and urine tests, to monitor the safety and effectiveness of the treatment.

Each clinical trial is different, and researchers must share all possible risks before you can consent to participate. Some possible risks include:

• Side effects from the medications or treatments used in the study
• Unwanted events may or may not relate to the study’s drug
• The treatment not working

The research team will monitor your health throughout the trial, regardless of whether you’re receiving the treatment or a placebo.

We’ve made it easy. By joining the ADPKD Registry or signing up for ACT (Accelerating Clinical Trials) Alerts, you’ll be able to determine your eligibility and be notified of clinical studies in your area.

Even if you’re unable to participate in a clinical trial, you can still support PKD research. Consider donating your PKD-affected kidneys for research when you undergo a nephrectomy, or surgical removal of non-functioning kidneys.

You can also share ongoing studies with friends or family who may qualify.

Tissue Donation

How do researchers make breakthroughs in PKD research? It’s often with the help of donated cystic kidney tissue.

Polycystic kidneys retrieved for research at the time of nephrectomy (surgical removal of non-functioning kidneys) provide an opportunity for scientists to study the cells and tissues that contain the genetic mutations responsible for cyst formation.

Our PKD tissue donation program provides patients with an avenue to contribute to the advancement of our understanding of PKD. We coordinate donations of discarded human PKD kidneys to research labs across the country, making it easy for you to make a difference.

As of April 2022, the PKD Foundation is able to connect patients with both autosomal dominant and autosomal recessive PKD with researchers collecting human tissue samples. Researchers are also interested in liver donations from ARPKD patients.

Reasons to Donate

One of the most valuable insights tissue donation provides researchers is the opportunity to study PKD in human cells.

So, tissue donation is one of the most important things patients can do to support PKD research.

There are so many reasons to donate your kidney tissue:

1. ADPKD cells harbor the genetic mutations that are responsible for cyst formation. ARPKD cells also provide valuable genetic information about disease progression.
2. It’s very difficult to replicate human disease in animals.
3. Medical research using human samples benefits future patients.
4. There is no cost to the patient to donate discarded PKD kidneys for research.
5. Your decision to donate does not impact your care.
6. Tissue donation is anonymous.

Your patient identifiers are removed at the hospital prior to shipping the tissues to the lab.

The only information provided about you is age, gender, prior dialysis and/or transplant status, blood urea nitrogen, creatinine levels, and details about your procedure.

The Donation Process

If your kidney(s) are going to be removed and you wish to donate them to PKD research, please follow these steps:

1. Advise your surgeon, nurse coordinator, and anyone else at the hospital of your wish to donate.
2. Complete these forms:
   Tissue donation information form
   ADPKD consent form
   ARPKD consent form
3. Contact us at (800) 753-2873 or by email at research@pkdcure.org.
   Important Note: Notify us and submit the forms at least two weeks before your surgery to allow time to coordinate the donation.

Absolutely!

Every advance we’ve made in treating this disease has been possible thanks to volunteer participants.

To develop better PKD treatments, researchers rely on the participation of patients like you!