New pill targets genetic form of polycystic kidney disease

NCT ID NCT07161037

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

First seen Jun 24, 2026 · Last updated Aug 12, 2026 · Updated 5 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.

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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

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