Living with Autosomal
Dominant Polycystic
Kidney Disease (ADPKD)?

SEE IF YOU QUALIFY
ABOUT THE ACTION CLINICAL RESEARCH STUDY

This clinical research study is testing the effectiveness and safety of the study drug, lixivaptan, for qualified adults living with Autosomal Dominant Polycystic Kidney Disease.

STUDY LOCATIONS

Find a participating site location near you and take a questionnaire to determine your eligibility.

Find out if you qualify for this study.

Fill out the form to determine if you may be eligible.

*Indicates a required field

Are you between the ages of 18 and 60?*
Have you been diagnosed with Autosomal Dominant Polycystic Kidney Disease?*
Are you currently pregnant, or trying to become pregnant?*
I agree to be contacted about future studies.
I agree to the Privacy Policy and agree to be contacted about this study, via phone, text, or email.*

ABOUT THE ACTION STUDY

The ACTION study is designed to assess how effective and safe the study drug, lixivaptan, is compared to a placebo (drug with no active ingredient) as a possible treatment for adults living with Autosomal Dominant Polycystic Kidney Disease (ADPKD).

ABOUT AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE (ADPKD)

ADPKD is a genetic disorder where the kidneys develop fluid-filled cysts that, in time, make the kidneys larger. The disorder is caused by a mutation of the PKD1 or PKD2 gene, interrupting normal cell activities in the kidney causing cysts to form and grow. As cysts grow in the kidney, normal kidney activities fail, and eventually require dialysis or a transplant.

The current treatment options for most ADPKD patients are limited to therapies that treat the effects of the disorder rather than the disorder itself. There is a drug that was approved by the FDA called tolvaptan or more commonly known as JYNARQUE® or JINARC® which addresses the disorder. Tolvaptan (JYNARQUE® or JINARC®) is also approved in other countries to slow the growth of cysts in ADPKD patients. However, according to FDA approved labeling, JYNARQUE® can cause serious liver problems.

A new form of treatment is needed to improve the quality of life and better manage this disorder which is why we are conducting the ACTION study.

*Cornec-LeGall E_Alam A_Perrone RD. Autosomal dominant polycystic kidney disease. Lancet 393:919;2019.

ABOUT THE STUDY DRUG, LIXIVAPTAN

The study drug that is being tested is called lixivaptan. It is an oral drug that works by blocking a receptor in the kidney that is known to cause cysts in ADPKD patients to grow. Lixivaptan is in the same drug class and has the same mechanism of action as tolvaptan.

Side effects of lixivaptan in past clinical studies were anticipated from the drug’s pharmacologic activities, e.g., signs and symptoms of increased water excretion such as dry mouth, thirst, and frequent urination. Similarly, side effects noted in the Phase 2 program in ADPKD subjects are consistent with the pharmacologic effects of the drug.

The above information references Wang X, Constans MM, Chebib FT, Torres VE, Pellegrini L. Effect of a vasopressin V2 receptor antagonist on polycystic kidney disease development in a rat model. Am J Nephrol 49:487;2019.

WHAT TO EXPECT IN THE ACTION STUDY

INFORMED CONSENT

To participate in this study, patients will be asked to read and sign an Informed Consent form (ICF). The form explains:

  • The purpose of the study
  • Study visits and procedures
  • Potential risks and benefits
  • Confidentiality
  • How your personally identifiable information will be used
  • Information on how to withdraw from the study

STUDY DETAILS

Approximately 1,200 qualifying adults will take part in the ACTION study in countries around the world. The ACTION study is designed to assess how effective and safe the study drug, lixivaptan, is compared to a placebo* as a possible treatment for adults living with ADPKD. In Part 1, patients enrolled in the study will be randomly assigned by a computer (as by chance, like the flip of a coin) to either the lixivaptan group or the placebo group. Patients will have 2 chances out of 3 to receive lixivaptan and 1 chance out of 3 to receive placebo. During Part 2 of this study, all enrolled participants will receive lixivaptan. During the study, there will be times where neither you nor the study team will know which treatment you are receiving.

*A placebo has no active ingredient and is used to compare the effects of the study drug.

VISIT PROCEDURES

As safety is our top priority, all enrolled patients will be monitored and supported by the study team. Procedures to make sure patients are doing well will be required in this study, some of them are:

  • Health and medication questions
  • Vital signs
  • Measurement of weight and height
  • Physical examinations
  • Blood and urine tests
  • Electrocardiogram (ECG)
  • Magnetic Resonance Imaging (MRI)
  • Health questionnaires

INFORMED CONSENT

To participate in this study, patients will be asked to read and sign an Informed Consent form (ICF). The form explains:

  • The purpose of the study
  • Study visits and procedures
  • Potential risks and benefits
  • Confidentiality
  • How your personally identifiable information will be used
  • Information on how to withdraw from the study

STUDY DETAILS

Approximately 1,200 qualifying adults will take part in the ACTION study in countries around the world. The ACTION study is designed to assess how effective and safe the study drug, lixivaptan, is compared to a placebo* as a possible treatment for adults living with ADPKD. In Part 1, patients enrolled in the study will be randomly assigned by a computer (as by chance, like the flip of a coin) to either the lixivaptan group or the placebo group. Patients will have 2 chances out of 3 to receive lixivaptan and 1 chance out of 3 to receive placebo. During Part 2 of this study, all enrolled participants will receive lixivaptan. During the study, there will be times where neither you nor the study team will know which treatment you are receiving.

*A placebo has no active ingredient and is used to compare the effects of the study drug.

VISIT PROCEDURES

As safety is our top priority, all enrolled patients will be monitored and supported by the study team. Procedures to make sure patients are doing well will be required in this study, some of them are:

  • Health and medication questions
  • Vital signs
  • Measurement of weight and height
  • Physical examinations
  • Blood and urine tests
  • Electrocardiogram (ECG)
  • Magnetic Resonance Imaging (MRI)
  • Health questionnaires

SCREENING

During screening, patients will be evaluated to see if they meet the study criteria. The screening period can last up to 8 weeks.

STUDY LENGTH

Enrolled participants will be asked to attend approximately 45 study visits for about 2.5 years. At some locations, some visits may be conducted at home by a home healthcare clinician (HHC).

FREQUENTLY ASKED QUESTIONS

What is a clinical research study?

Before a drug or treatment can be approved by regulatory authorities for use, there must be proof that the investigational study drug works and is considered safe enough for the tested population. Clinical research studies, also called clinical trials, look for new ways to prevent, detect, and treat disease. They are designed to test the safety and effectiveness of investigational study drugs to understand if these potential treatments can be used in the future.

Will compensation be provided?

Study-related therapies and assessments will be provided at no cost. Participants will not be paid to take part in this study; however, reasonable study-related expenses may be reimbursed.

What happens to personal information?

There are security measures to protect personal information. This is to avoid personal information from being lost, used, altered, disclosed, or accessed in any unauthorized way. To protect the identity of the participant, personal study information will be coded (e.g., unique study numbers are assigned to each patient). More information about privacy maintenance will be shared with those who choose to participate in the study.

Can participants withdraw from the clinical trial after they’ve started?

Taking part in a clinical research study is entirely voluntary. Participants can withdraw at any time for any reason. Care by doctors and nurses will not change.

Who is sponsoring this clinical research study?

Palladio Biosciences, Inc. a clinical stage biopharmaceutical company, is sponsoring this clinical research study. For more information about Palladio, please visit www.palladiobio.com.

How can I learn more about the study drug and this study?

More information on this clinical research study can be found at ClinicalTrials.gov (NCT04064346) and information on the study drug can be found on the Palladio website.

Who oversees clinical research studies?

To help ensure that a research study is ethical, and that participants’ rights are protected, Institutional Review Boards (IRB), Ethics Committees (EC) or Ministry of Health (MOH) review and approve study protocols that detail all aspects of how the study is conducted. There are also teams that monitor the study called Clinical Research Associates; they visit the study sites to make sure the study protocol procedures, research requirements and country regulations are being followed throughout the study. Health authorities may also inspect the study sites to ensure the safety and rights of the participants in a trial and the integrity of the research study objectives.

What is an investigational study drug?

An investigational study drug is a product that has not been approved by a country’s regulatory authorities (e.g. Food and Drug Administration in the U.S., European Medicines Agency in Europe, or your country’s regulatory agency for new drugs) for medical use. The product must go through a clinical research study to assess the effectiveness and safety.

What is a placebo?

A placebo has no active ingredient and is used to compare the effects of the study drug. The placebo will look like the study drug and will be administered the same way to make sure that no one, not even the study doctor, will know which drug has been assigned to whom unless the information is needed for your safety.

STUDY LOCATIONS

The ACTION study will be taking place in countries all over the world. Current study locations are listed below. Those highlighted in green are currently open and those highlighted in red will be open soon. If you are interested in participating, click “See if you qualify” and complete the questionnaire to find out if you may be eligible for the study.

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