NCT07161037

Brief 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.

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at below P25 for phase_2

Timeline
20mo left

Started Nov 2025

Geographic Reach
7 countries

40 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress22%
Nov 2025Dec 2027

First Submitted

Initial submission to the registry

August 29, 2025

Completed
10 days until next milestone

First Posted

Study publicly available on registry

September 8, 2025

Completed
2 months until next milestone

Study Start

First participant enrolled

November 19, 2025

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 22, 2027

Expected
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2027

Last Updated

April 24, 2026

Status Verified

November 1, 2025

Enrollment Period

1.7 years

First QC Date

August 29, 2025

Last Update Submit

April 21, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • htTKV on MRI Over Time

    Baseline up to End of Study (Week 52)

Secondary Outcomes (3)

  • Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    From Day 1 up to Week 54

  • Maximum Observed Plasma Concentration (Cmax) of VX-407

    From Day 1 up to Week 52

  • Area Under the Concentration Versus Time Curve (AUC) of VX-407

    From Day 1 up to Week 52

Study Arms (1)

VX-407

EXPERIMENTAL

Participants will receive VX-407 for up to 52 weeks.

Drug: VX-407

Interventions

VX-407DRUG

Tablets for oral administration.

VX-407

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (40)

Alabama Kidney Research

Alabaster, Alabama, 35007, United States

RECRUITING

UAB Nephrology Research at CH19

Birmingham, Alabama, 35294, United States

RECRUITING

Kaiser Permanente Los Angeles Medical Center

Los Angeles, California, 90027, United States

RECRUITING

UCSF Clinical Research Center at Parnassus

San Francisco, California, 94143, United States

RECRUITING

Nephrology & Hypertension Associates, PC

Middlebury, Connecticut, 06762, United States

RECRUITING

Yale University - Yale Nephrology Clinical Research Clinic

New Haven, Connecticut, 06510, United States

RECRUITING

Mayo Clinic - Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Emory School of Medicine - Renal Division

Atlanta, Georgia, 30322, United States

RECRUITING

Renal Associates

Columbus, Georgia, 31904, United States

RECRUITING

UChicago Medicine - Duchossois Center for Advanced Medicine - Nephrology

Chicago, Illinois, 60637, United States

RECRUITING

University of Iowa Health Care Medical Center- Nephrology

Iowa City, Iowa, 52242, United States

RECRUITING

Clinical & Translational Science Unit (CTSU) - Nephrology

Kansas City, Kansas, 66160, United States

RECRUITING

University of Maryland Medical Center - General Clinical Research Center

Baltimore, Maryland, 21201, United States

RECRUITING

Tufts Medical Center

Boston, Massachusetts, 02111, United States

RECRUITING

Beth Israel Deaconess Medical Center - Nephrology

Boston, Massachusetts, 02215, United States

RECRUITING

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

Minneapolis, Minnesota, 55455, United States

RECRUITING

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

DaVita Kidney Care - Las Vegas

Las Vegas, Nevada, 89107, United States

RECRUITING

The Rogosin Kidney Center

New York, New York, 10021, United States

RECRUITING

General Nephrology Clinic - New York City

New York, New York, 10032, United States

RECRUITING

Cleveland Clinic - Main Campus

Cleveland, Ohio, 44195, United States

RECRUITING

Geisinger Clinic

Danville, Pennsylvania, 17822, United States

RECRUITING

Perelman Center for Advanced Medicine - Nephrology

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

UVM Medical Center - 1 South Prospect Street

Burlington, Vermont, 05401, United States

RECRUITING

Swedish Center for Comprehensive Care

Seattle, Washington, 98104, United States

RECRUITING

Universite Catholique de Louvain - Nephrology

Brussels, Belgium

RECRUITING

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Nephrology

Leuven, Belgium

RECRUITING

McGill University Health Centre

Montreal, Canada

RECRUITING

Centre Hospitalier Regional Universitaire (CHRU) Brest

Brest, France

RECRUITING

Hopital Necker Enfants Malades - Neurology

Paris, France

RECRUITING

Charité - Klinik für Nephrologie und Intensivmedizin der

Berlin, Germany

RECRUITING

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

Cologne, Germany

RECRUITING

Fundacio Puigvert

Barcelona, Spain

RECRUITING

Hospital Fundacion Jimenez Diaz

Madrid, Spain

RECRUITING

Queen Elizabeth Hospital Birmingham

Birmingham, United Kingdom

RECRUITING

King's College Hospital - Pulmonology

London, United Kingdom

RECRUITING

Royal Free Hospital - Nephrology

London, United Kingdom

RECRUITING

St George's Hospital London - Nephrology

London, United Kingdom

RECRUITING

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

Newcastle upon Tyne, United Kingdom

RECRUITING

Northern General Hospital

Sheffield, United Kingdom

RECRUITING

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Condition Hierarchy (Ancestors)

Polycystic Kidney DiseasesKidney Diseases, CysticKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCiliopathiesGenetic Diseases, Inborn

Central Study Contacts

Medical Information

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 29, 2025

First Posted

September 8, 2025

Study Start

November 19, 2025

Primary Completion (Estimated)

July 22, 2027

Study Completion (Estimated)

December 31, 2027

Last Updated

April 24, 2026

Record last verified: 2025-11

Data Sharing

IPD Sharing
Will not share

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/

Locations