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 (38) 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
UAB Nephrology Research at CH19
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
Clinical & Translational Science Unit (CTSU) - 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
General Nephrology Clinic - New York City
RECRUITING
New York
Ohio
Cleveland Clinic - Main Campus
RECRUITING
Cleveland
Pennsylvania
Geisinger Clinic
RECRUITING
Danville
Perelman Center for Advanced Medicine - Nephrology
RECRUITING
Philadelphia
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
Hopital Necker Enfants Malades - Neurology
RECRUITING
Paris
Germany
Charité - Klinik für Nephrologie und Intensivmedizin der
RECRUITING
Berlin
University Hospital Cologne (Koeln) - Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin
RECRUITING
Cologne
Spain
Fundacio Puigvert
RECRUITING
Barcelona
Hospital Fundacion Jimenez Diaz
RECRUITING
Madrid
United Kingdom
Queen Elizabeth Hospital Birmingham
RECRUITING
Birmingham
King's College Hospital - Pulmonology
RECRUITING
London
Royal Free Hospital - Nephrology
RECRUITING
London
St George's Hospital London - 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