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New pill targets genetic form of polycystic kidney disease

NCT ID NCT07161037

Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Sep 17, 2026 · Updated 7 times

Summary

This study tests an experimental drug called VX-407 in 24 adults with autosomal dominant polycystic kidney disease (ADPKD) who have specific changes in the PKD1 gene. The goal is to see if the drug can slow kidney growth and is safe. Participants take VX-407 tablets by mouth and undergo MRI scans and blood tests over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
VX-407 (tablet)
What this could lead to
If successful, this could point toward a treatment that slows kidney growth and preserves kidney function in people with a specific genetic form of polycystic kidney disease.
What could go wrong
This is an early Phase 2a study with only 24 participants, so results may not apply to everyone. The drug targets only certain PKD1 gene variants, and safety and effectiveness are still unproven.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

26 people

The number who actually took part.

Started

Nov 2025

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * A pre-existing diagnosis of ADPKD as defined in the protocol * Willing and able to comply with scheduled visits and other study procedures * Participants with ADPKD with Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E confirmed by abdominal MRI obtained during screening * Estimated glomerular filtration rate (eGFR) greater than or equal to (≥) 25 milliliter per minute (mL/min)/1.73 square meter (m\^2) Key Exclusion Criteria: * History of kidney disease other than ADPKD that in the opinion of the investigator would independently impact the natural history of ADPKD * History of solid organ or bone marrow transplantation or nephrectomy * Ongoing renal replacement therapy or planning to start renal replacement therapy within 12 months of screening Other protocol defined Inclusion/Exclusion criteria will apply.

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Conditions

The condition(s) this trial relates to.

autosomal dominant polycystic kidney disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Autosomal dominant polycystic kidney disease (adpkd)

Contacts and locations

Locations

  • Alabama Kidney Research

    Alabaster, Alabama, 35007, United States

  • Beth Israel Deaconess Medical Center - Nephrology

    Boston, Massachusetts, 02215, United States

  • Centre Hospitalier Regional Universitaire (CHRU) Brest

    Brest, France

  • Charité - Klinik für Nephrologie und Intensivmedizin der

    Berlin, Germany

  • Cleveland Clinic - Main Campus

    Cleveland, Ohio, 44195, United States

  • Clinical & Translational Science Unit (CTSU) - Nephrology

    Kansas City, Kansas, 66160, United States

  • DaVita Kidney Care - Las Vegas

    Las Vegas, Nevada, 89107, United States

  • Emory School of Medicine - Renal Division

    Atlanta, Georgia, 30322, United States

  • Fundacio Puigvert

    Barcelona, Spain

  • Geisinger Clinic

    Danville, Pennsylvania, 17822, United States

  • General Nephrology Clinic - New York City

    New York, New York, 10032, United States

  • Hopital Bretonneau

    Tours, France

  • Hopital Necker Enfants Malades - Neurology

    Paris, France

  • Hospital Fundacion Jimenez Diaz

    Madrid, Spain

  • Kaiser Permanente Los Angeles Medical Center

    Los Angeles, California, 90027, United States

  • King's College Hospital - Pulmonology

    London, United Kingdom

  • Mayo Clinic - Jacksonville

    Jacksonville, Florida, 32224, United States

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905, United States

  • McGill University Health Centre

    Montreal, Canada

  • Nephrology & Hypertension Associates, PC

    Middlebury, Connecticut, 06762, United States

  • Nephrology Clinical Trials Center

    Nashville, Tennessee, 37232, United States

  • Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital - Nephrology

    Newcastle upon Tyne, United Kingdom

  • Northern General Hospital

    Sheffield, United Kingdom

  • Perelman Center for Advanced Medicine - Nephrology

    Philadelphia, Pennsylvania, 19104, United States

  • Queen Elizabeth Hospital Birmingham

    Birmingham, United Kingdom

  • Renal Associates

    Columbus, Georgia, 31904, United States

  • Royal Free Hospital - Nephrology

    London, United Kingdom

  • St George's Hospital London - Nephrology

    London, United Kingdom

  • Swedish Center for Comprehensive Care

    Seattle, Washington, 98104, United States

  • The Rogosin Kidney Center

    New York, New York, 10021, United States

  • Tufts Medical Center

    Boston, Massachusetts, 02111, United States

  • UAB Nephrology Research at CH19

    Birmingham, Alabama, 35294, United States

  • UCSF Clinical Research Center at Parnassus

    San Francisco, California, 94143, United States

  • UChicago Medicine - Duchossois Center for Advanced Medicine - Nephrology

    Chicago, Illinois, 60637, United States

  • UMCG - Nephrology

    Groningen, Netherlands

  • UVM Medical Center - 1 South Prospect Street

    Burlington, Vermont, 05401, United States

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Nephrology

    Leuven, Belgium

  • Universite Catholique de Louvain - Nephrology

    Brussels, Belgium

  • University Hospital Cologne (Koeln) - Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin

    Cologne, Germany

  • University of Iowa Health Care Medical Center- Nephrology

    Iowa City, Iowa, 52242, United States

  • University of Maryland Medical Center - General Clinical Research Center

    Baltimore, Maryland, 21201, United States

  • University of Minnesota -Clinics and Surgery Center (CSC) - Endocrine Clinic

    Minneapolis, Minnesota, 55455, United States

  • Yale University - Yale Nephrology Clinical Research Clinic

    New Haven, Connecticut, 06510, United States

More trials for these conditions

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