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
Vertex Pharmaceuticals Incorporated
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
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: * 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) Key Exclusion Criteria: * History of kidney disease other than ADPKD that in the opinion of the investigator would independently…
Interventions
- DrugVX-407
Tablets for oral administration.
Locations (40)
- Alabama Kidney ResearchAlabaster, Alabama
- UAB Nephrology Research at CH19Birmingham, Alabama
- Kaiser Permanente Los Angeles Medical CenterLos Angeles, California
- UCSF Clinical Research Center at ParnassusSan Francisco, California
- Nephrology & Hypertension Associates, PCMiddlebury, Connecticut
- Yale University - Yale Nephrology Clinical Research ClinicNew Haven, Connecticut