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A Study Testing VX-407 in Patients with Autosomal Dominant Polycystic Kidney Disease Who Have Specific PKD1 Gene Changes

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

Autosomal Dominant Polycystic Kidney Disease is a condition that runs in families where many fluid-filled sacs called cysts grow in the kidneys over time. These cysts can make the kidneys larger and may affect how well they work. This study is looking at a medication called VX-407 given as a tablet by mouth. The purpose of the study is to see if this treatment can help people with this kidney disease who have certain specific changes in a gene called PKD1. The study will also look at whether the medication is safe and how the body processes it.

During the study, participants will take VX-407 for up to 52 weeks. The main focus is to measure changes in the total size of the kidneys using a type of scan called MRI, which takes detailed pictures of the inside of the body. The study will check if the medication can slow down or stop the growth of the kidneys compared to the beginning of the study. Doctors will also watch for any side effects and will regularly check blood tests, heart tracings using a test called ECG, and other health measurements to make sure the medication is safe.

Before joining the study, participants will have a special genetic test done using a device that looks for specific changes in the PKD1 gene. This test uses a blood sample and looks for particular types of genetic changes that are not the kind that completely stop the gene from working. Only people with these specific genetic changes and who have kidneys of a certain size based on the MRI scan will be included in the study.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment and confirmation

    Upon joining the study, your eligibility will be confirmed. This includes verification that you are between 18 and 65 years of age and have a body mass index (a measure of body fat based on height and weight) between 18 and 45 kg/m².

    Your diagnosis of autosomal dominant polycystic kidney disease (a genetic condition causing fluid-filled sacs called cysts to grow in the kidneys) will be verified through existing medical records and imaging results.

    An abdominal MRI scan (a type of imaging that uses magnetic fields to create detailed pictures of internal organs) will be performed to measure your total kidney volume and confirm your classification status.

    Your kidney function will be assessed through blood tests to ensure your estimated glomerular filtration rate (a measure of how well your kidneys filter waste from blood) is at least 25 ml/min/1.73 m².

    A genetic test using a designated study assay will confirm that you have a specific type of gene variant in the PKD1 gene (the gene associated with your condition) that qualifies you for this study.

  2. Step 2

    Treatment period with VX-407

    You will receive VX-407, which is provided as a tablet to be taken by mouth.

    The study will monitor how the medication affects your kidney cyst growth over time.

    Regular blood samples will be collected to measure the levels of VX-407 in your bloodstream and to assess how your body processes the medication.

  3. Step 3

    Ongoing monitoring and assessments

    Throughout the study, repeated MRI scans will be performed to measure changes in your total kidney volume compared to the initial measurement.

    Regular safety assessments will be conducted, including monitoring for any adverse events (unwanted or harmful effects that may occur during treatment).

    Blood tests will be performed regularly to check your blood cell counts, kidney and liver function, and blood clotting ability.

    Electrocardiograms (tests that measure the electrical activity of your heart, abbreviated as ECGs) will be performed using a standard 12-lead setup.

    Your vital signs (measurements such as blood pressure, heart rate, and temperature) will be monitored at scheduled visits.

  4. Step 4

    Study completion

    The study is expected to continue until approximately August 2027.

    Final assessments will include measurement of kidney volume changes, safety evaluations, and collection of information about how the medication worked in your body.

Who can join the trial?

7 criteria

  • You must be between 18 and 65 years old at the time of signing the consent form for the screening period
  • Your body mass index (a measure of body fat based on height and weight) must be between 18 and 45 kg/m2
  • You must have a confirmed diagnosis of Autosomal Dominant Polycystic Kidney Disease (a genetic condition where fluid-filled sacs called cysts grow in the kidneys), which can be shown by:
    • Having evidence that one or both of your biological parents have polycystic kidney disease AND having a certain number of cysts in your kidneys based on your age: at least 3 cysts total in both kidneys if you are younger than 40 years old, at least 2 cysts in each kidney if you are between 40 and 59 years old, or at least 4 cysts in each kidney if you are 60 years old or older
    • OR if you do not have a family history of this disease: having at least 10 cysts in each kidney
    • You must have a specific Mayo imaging classification status (a system that measures kidney size and disease progression) of 1B with a kidney volume of at least 250 mL/m, or 1C, 1D, or 1E, confirmed by an MRI scan (a type of imaging test) of your abdomen done during the screening period
    • You must have a specific type of genetic variant (a change in your genes) in the PKD1 gene that is disease-causing, likely disease-causing, or uncertain, but not a type that stops the gene from working completely. This must be confirmed by a special genetic test
    • Your estimated kidney function (a measure of how well your kidneys filter waste from your blood) must be at least 25 mL/min/1.73 m2 based on a specific calculation method

Who cannot join the trial?

1 criterion

  • No specific exclusion criteria have been provided for this clinical trial at this time
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Investigated drugs

VX-407 is an investigational medication being studied for the treatment of autosomal dominant polycystic kidney disease (ADPKD). This is a genetic condition where fluid-filled cysts grow in the kidneys and can cause them to lose function over time. VX-407 is being tested specifically in patients who have certain variants in a gene called PKD1, which is responsible for causing this form of kidney disease. The medication is designed to help slow down or stop the progression of the disease.

What is already known about the treatment

VX-407 – VX-407 is an experimental medication currently being studied in Phase 2a clinical trials for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), specifically in patients who have certain variations in the PKD1 gene. ADPKD is an inherited condition where fluid-filled cysts grow in the kidneys, gradually reducing their ability to function properly. While the exact way VX-407 works at the molecular level has not been fully disclosed in this trial information, it is being investigated as a potential treatment to slow down or stop the progression of cyst formation in the kidneys. The medication is being tested in an open-label study to evaluate how well it works, its safety profile, and how the body processes it when given to patients with this specific genetic form of kidney disease.

Investigated diseases

Autosomal Dominant Polycystic Kidney Disease – Autosomal Dominant Polycystic Kidney Disease is a genetic disorder that affects the kidneys and is passed down through families. The condition causes numerous fluid-filled sacs called cysts to develop in both kidneys over time. These cysts gradually grow larger and increase in number as a person ages. As the cysts expand, they replace normal kidney tissue and cause the kidneys to enlarge significantly. The growing cysts interfere with the kidneys' ability to filter waste products from the blood and maintain proper fluid balance in the body. Over many years, the progressive enlargement and multiplication of cysts leads to reduced kidney function.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-517393-13-00Protocol codeVX24-407-101Estimated enrolment24 patientsSponsorVertex Pharmaceuticals Inc.

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