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Study on the Effects of Inaxaplin for Adults and Children with APOL1-mediated Proteinuric Kidney Disease

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What is this trial about?

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

This clinical trial is focused on studying a condition known as APOL1-mediated Proteinuric Kidney Disease, which affects the kidneys and can lead to protein loss in the urine. The trial is testing a new treatment called VX-147, which is taken as a film-coated tablet. The purpose of the study is to evaluate how effective and safe VX-147 is for people with this kidney disease.

Participants in the study will receive either the VX-147 tablet or a placebo tablet. The study is designed to be adaptive, meaning it can change based on how participants respond to the treatment. It is also double-blind, so neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure that the results are unbiased. The study will include both adults and children, and it will take place over a period of time to monitor the effects of the treatment.

The trial will assess various outcomes, such as changes in kidney function and the safety of the treatment. Participants will have regular check-ups and tests to monitor their health and the impact of the medication. The study aims to provide valuable information about the potential benefits of VX-147 for those with APOL1-mediated Proteinuric Kidney Disease.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant will sign an informed consent form. This document confirms understanding of the study and agreement to participate.

    Participants must be willing and able to attend scheduled visits, follow the treatment plan, and adhere to study guidelines.

  2. Step 2

    Initial assessment

    Participants will undergo an initial assessment to confirm eligibility. This includes genetic testing for the APOL1 genotype and other health evaluations.

    Participants must have a body mass index (BMI) between 18 and 45 kg/m² and a total body weight of at least 40 kg.

  3. Step 3

    Treatment phase

    Participants will receive either the VX-147 film-coated tablet or a placebo tablet. The medication is taken orally.

    The dosage and frequency will be determined by the study protocol, and participants will be monitored regularly for safety and effectiveness.

  4. Step 4

    Regular monitoring

    Participants will attend regular visits to monitor health and response to the treatment. This includes laboratory tests and health assessments.

    The study will evaluate changes in kidney function and other health indicators over time.

  5. Step 5

    Completion of study

    The study is expected to last until May 2028. Participants will be informed of their progress and any findings related to their health.

    Upon completion, participants may receive information about the study results and any potential next steps.

Who can join the trial?

10 criteria

  • The person or their legal representative must sign and date a consent form to participate in the study.
  • The person must be willing and able to attend scheduled visits, follow the treatment plan, and comply with study guidelines and procedures.
  • The person must have a specific genetic type called APOL1 genotype, which can be G1/G1, G2/G2, or G1/G2, confirmed by a special test.
  • For Phase 2 of the study, the person must be between 18 and 65 years old. For Phase 3, they must be between 12 and 65 years old. No one over 65 can join the study.
  • The person must have a Body Mass Index (BMI) between 18 and 45 and weigh at least 40 kg (about 88 pounds).
  • The person must have a specific range of protein in their urine, measured in the morning over three days.
  • The person must have a certain level of kidney function, measured by a test called estimated glomerular filtration rate (eGFR).
  • The person must be on a stable dose of a medication called an angiotensin converting enzyme (ACE) inhibitor or an angiotensin receptor blocker (ARB) for at least four weeks before the study, unless they cannot tolerate these medications.
  • If the person is taking other specific medications like sodium glucose co-transporter-2 (SGLT2) inhibitors, mineralocorticoid receptor antagonists (MRAs), or certain immunosuppressants, they must be on a stable dose for four weeks before the study.
  • The person must be part of a social security scheme.

Who cannot join the trial?

7 criteria

  • Patients with any other serious medical condition that might affect their participation in the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a kidney transplant.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who have a known allergy to the study medication or its ingredients.
  • Patients who are unable to comply with the study procedures.
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Investigated drugs

VX-147 is a medication being studied for its potential to help people with a specific type of kidney disease called APOL1-mediated proteinuric kidney disease. This condition affects the kidneys and can lead to protein leaking into the urine, which is not normal. The goal of using VX-147 in this trial is to see if it can reduce the amount of protein in the urine and improve kidney function. Researchers are also looking at how safe the medication is for both adults and children who have this kidney disease. By participating in this trial, patients may help determine if VX-147 can be an effective treatment for their condition.

What is already known about the treatment

VX-147 – VX-147 is administered orally and is currently being studied in clinical trials for its effectiveness and safety in treating APOL1-mediated Proteinuric Kidney Disease (AMKD). This medication is in the experimental phase, specifically in a Phase 2/3 trial, and is not yet widely recognized in medical literature as a standard treatment. The main therapeutic indication for VX-147 is to address kidney disease associated with the APOL1 gene, which can lead to protein leakage in the urine. At the molecular level, VX-147 works by targeting specific pathways involved in kidney function to reduce proteinuria, which is the presence of excess protein in the urine. It is classified pharmacologically as a potential treatment for kidney-related conditions.

Investigated diseases

APOL1-mediated Proteinuric Kidney Disease (AMKD) – This disease is characterized by the presence of protein in the urine, which indicates kidney damage. It is associated with genetic variations in the APOL1 gene, which can lead to kidney dysfunction. Over time, the kidneys' ability to filter waste from the blood diminishes, resulting in a decline in kidney function. The progression of the disease can lead to a significant reduction in the estimated glomerular filtration rate (eGFR), a key measure of kidney health. As the disease advances, it may result in end-stage kidney disease (ESKD), where the kidneys can no longer function adequately. This condition is considered rare and primarily affects individuals with certain genetic backgrounds.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-515633-15-00Protocol codeVX21-147-301Estimated enrolment466 patientsSponsorVertex Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

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