RecruitingPhase 2NCT07161037

Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)

A Phase 2a, Open-label, Single-arm Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-407 in Subjects With Autosomal Dominant Polycystic Kidney Disease Who Have a Subset of PKD1 Gene Variants


Sponsor

Vertex Pharmaceuticals Incorporated

Enrollment

24 participants

Start Date

Nov 19, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

The purpose of the study is to evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV), safety, tolerability, and pharmacokinetics (PK) of VX-407.


Eligibility

Min Age: 18 YearsMax Age: 65 Years

Inclusion Criteria4

  • 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)

Exclusion Criteria3

  • 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

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Interventions

DRUGVX-407

Tablets for oral administration.


Locations(40)

Alabama Kidney Research

Alabaster, Alabama, United States

UAB Nephrology Research at CH19

Birmingham, Alabama, United States

Kaiser Permanente Los Angeles Medical Center

Los Angeles, California, United States

UCSF Clinical Research Center at Parnassus

San Francisco, California, United States

Nephrology & Hypertension Associates, PC

Middlebury, Connecticut, United States

Yale University - Yale Nephrology Clinical Research Clinic

New Haven, Connecticut, United States

Mayo Clinic - Jacksonville

Jacksonville, Florida, United States

Emory School of Medicine - Renal Division

Atlanta, Georgia, United States

Renal Associates

Columbus, Georgia, United States

UChicago Medicine - Duchossois Center for Advanced Medicine - Nephrology

Chicago, Illinois, United States

University of Iowa Health Care Medical Center- Nephrology

Iowa City, Iowa, United States

Clinical & Translational Science Unit (CTSU) - Nephrology

Kansas City, Kansas, United States

University of Maryland Medical Center - General Clinical Research Center

Baltimore, Maryland, United States

Tufts Medical Center

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center - Nephrology

Boston, Massachusetts, United States

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

Minneapolis, Minnesota, United States

Mayo Clinic - Rochester

Rochester, Minnesota, United States

DaVita Kidney Care - Las Vegas

Las Vegas, Nevada, United States

The Rogosin Kidney Center

New York, New York, United States

General Nephrology Clinic - New York City

New York, New York, United States

Cleveland Clinic - Main Campus

Cleveland, Ohio, United States

Geisinger Clinic

Danville, Pennsylvania, United States

Perelman Center for Advanced Medicine - Nephrology

Philadelphia, Pennsylvania, United States

UVM Medical Center - 1 South Prospect Street

Burlington, Vermont, United States

Swedish Center for Comprehensive Care

Seattle, Washington, United States

Universite Catholique de Louvain - Nephrology

Brussels, Belgium

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Nephrology

Leuven, Belgium

McGill University Health Centre

Montreal, Canada

Centre Hospitalier Regional Universitaire (CHRU) Brest

Brest, France

Hopital Necker Enfants Malades - Neurology

Paris, France

Charité - Klinik für Nephrologie und Intensivmedizin der

Berlin, Germany

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

Cologne, Germany

Fundacio Puigvert

Barcelona, Spain

Hospital Fundacion Jimenez Diaz

Madrid, Spain

Queen Elizabeth Hospital Birmingham

Birmingham, United Kingdom

King's College Hospital - Pulmonology

London, United Kingdom

Royal Free Hospital - Nephrology

London, United Kingdom

St George's Hospital London - Nephrology

London, United Kingdom

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

Newcastle upon Tyne, United Kingdom

Northern General Hospital

Sheffield, United Kingdom

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NCT07161037


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