New pill targets genetic form of polycystic kidney disease

NCT ID NCT07161037

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Alabama Kidney Research
RECRUITING

Alabaster, Alabama, 35007, United States
Beth Israel Deaconess Medical Center - Nephrology
RECRUITING

Boston, Massachusetts, 02215, United States
Centre Hospitalier Regional Universitaire (CHRU) Brest
RECRUITING

Brest, France
Charité - Klinik für Nephrologie und Intensivmedizin der
RECRUITING

Berlin, Germany
Cleveland Clinic - Main Campus
RECRUITING

Cleveland, Ohio, 44195, United States
Clinical & Translational Science Unit (CTSU) - Nephrology
RECRUITING

Kansas City, Kansas, 66160, United States
DaVita Kidney Care - Las Vegas
RECRUITING

Las Vegas, Nevada, 89107, United States
Emory School of Medicine - Renal Division
RECRUITING

Atlanta, Georgia, 30322, United States
Fundacio Puigvert
RECRUITING

Barcelona, Spain
Geisinger Clinic
RECRUITING

Danville, Pennsylvania, 17822, United States
General Nephrology Clinic - New York City
RECRUITING

New York, New York, 10032, United States
Hopital Bretonneau
RECRUITING

Tours, France
Hopital Necker Enfants Malades - Neurology
RECRUITING

Paris, France
Hospital Fundacion Jimenez Diaz
RECRUITING

Madrid, Spain
Kaiser Permanente Los Angeles Medical Center
RECRUITING

Los Angeles, California, 90027, United States
King's College Hospital - Pulmonology
RECRUITING

London, United Kingdom
Mayo Clinic - Jacksonville
RECRUITING

Jacksonville, Florida, 32224, United States
Mayo Clinic - Rochester
RECRUITING

Rochester, Minnesota, 55905, United States
McGill University Health Centre
RECRUITING

Montreal, Canada
Nephrology & Hypertension Associates, PC
RECRUITING

Middlebury, Connecticut, 06762, United States
Nephrology Clinical Trials Center
RECRUITING

Nashville, Tennessee, 37232, United States
Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital - Nephrology
RECRUITING

Newcastle upon Tyne, United Kingdom
Northern General Hospital
RECRUITING

Sheffield, United Kingdom
Perelman Center for Advanced Medicine - Nephrology
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Queen Elizabeth Hospital Birmingham
RECRUITING

Birmingham, United Kingdom
Renal Associates
RECRUITING

Columbus, Georgia, 31904, United States
Royal Free Hospital - Nephrology
RECRUITING

London, United Kingdom
St George's Hospital London - Nephrology
RECRUITING

London, United Kingdom
Swedish Center for Comprehensive Care
RECRUITING

Seattle, Washington, 98104, United States
The Rogosin Kidney Center
RECRUITING

New York, New York, 10021, United States
Tufts Medical Center
RECRUITING

Boston, Massachusetts, 02111, United States
UAB Nephrology Research at CH19
RECRUITING

Birmingham, Alabama, 35294, United States
UCSF Clinical Research Center at Parnassus
RECRUITING

San Francisco, California, 94143, United States
UChicago Medicine - Duchossois Center for Advanced Medicine - Nephrology
RECRUITING

Chicago, Illinois, 60637, United States
UMCG - Nephrology
RECRUITING

Groningen, Netherlands
UVM Medical Center - 1 South Prospect Street
RECRUITING

Burlington, Vermont, 05401, United States
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Nephrology
RECRUITING

Leuven, Belgium
Universite Catholique de Louvain - Nephrology
RECRUITING

Brussels, Belgium
University Hospital Cologne (Koeln) - Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin
RECRUITING

Cologne, Germany
University of Iowa Health Care Medical Center- Nephrology
RECRUITING

Iowa City, Iowa, 52242, United States
University of Maryland Medical Center - General Clinical Research Center
RECRUITING

Baltimore, Maryland, 21201, United States
University of Minnesota -Clinics and Surgery Center (CSC) - Endocrine Clinic
RECRUITING

Minneapolis, Minnesota, 55455, United States
Yale University - Yale Nephrology Clinical Research Clinic
RECRUITING

New Haven, Connecticut, 06510, United States