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
Status: Recruiting
Location: See all (29) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• 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)
Locations
United States
Alabama
Alabama Kidney Research
RECRUITING
Alabaster
University of Alabama at Birmingham- Nephrology Research Clinic
RECRUITING
Birmingham
California
Kaiser Permanente Los Angeles Medical Center
RECRUITING
Los Angeles
UCSF Clinical Research Center at Parnassus
RECRUITING
San Francisco
Connecticut
Nephrology & Hypertension Associates, PC
RECRUITING
Middlebury
Yale University - Yale Nephrology Clinical Research Clinic
RECRUITING
New Haven
Florida
Mayo Clinic - Jacksonville
RECRUITING
Jacksonville
Georgia
Emory School of Medicine - Renal Division
RECRUITING
Atlanta
Renal Associates
RECRUITING
Columbus
Iowa
University of Iowa Health Care Medical Center- Nephrology
RECRUITING
Iowa City
Illinois
UChicago Medicine - Duchossois Center for Advanced Medicine - Nephrology
RECRUITING
Chicago
Kansas
The University of Kansas Medical Center Division of Nephrology
RECRUITING
Kansas City
Massachusetts
Beth Israel Deaconess Medical Center - Nephrology
RECRUITING
Boston
Tufts Medical Center
RECRUITING
Boston
Maryland
University of Maryland Medical Center - General Clinical Research Center
RECRUITING
Baltimore
Minnesota
University of Minnesota -Clinics and Surgery Center (CSC) - Endocrine Clinic
RECRUITING
Minneapolis
Mayo Clinic - Rochester
RECRUITING
Rochester
Nevada
DaVita Kidney Care - Las Vegas
RECRUITING
Las Vegas
New York
Columbia University Medical Center/NYP
RECRUITING
New York
Vermont
UVM Medical Center - 1 South Prospect Street
RECRUITING
Burlington
Other Locations
Belgium
Universite Catholique de Louvain - Nephrology
RECRUITING
Brussels
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Nephrology
RECRUITING
Leuven
Canada
McGill University Health Centre
RECRUITING
Montreal
France
Centre Hospitalier Regional Universitaire (CHRU) Brest
RECRUITING
Brest
United Kingdom
Queen Elizabeth Hospital Birmingham
RECRUITING
Birmingham
King's College Hospital - Pulmonology
RECRUITING
London
Royal Free Hospital - Nephrology
RECRUITING
London
Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital - Nephrology
RECRUITING
Newcastle Upon Tyne
Northern General Hospital
RECRUITING
Sheffield
Contact Information
Primary
Medical Information
medicalinfo@vrtx.com
617-341-6777
Time Frame
Start Date: 2025-11-19
Estimated Completion Date: 2027-12-31
Participants
Target number of participants: 24
Treatments
Experimental: VX-407
Participants will receive VX-407 for up to 52 weeks.
Related Therapeutic Areas
Sponsors
Leads: Vertex Pharmaceuticals Incorporated