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
Trial status: Authorised
Percent change from baseline in htTKV on MRI over time, Safety and tolerability of VX¬407 based on adverse events (AEs), clinical laboratory values (i.e., hematology, serum chemistry, coagulation), standard 12-lead ECGs, and vital signs, Plasma PK parameters of VX 407
Proportion of subjects with percent change from baseline in htTKV ≤0 on MRI over time
Description
Percent change from baseline in htTKV on MRI over time, Safety and tolerability of VX¬407 based on adverse events (AEs), clinical laboratory values (i.e., hematology, serum chemistry, coagulation), standard 12-lead ECGs, and vital signs, Plasma PK parameters of VX 407
Who can participate
Age group: 18-64 years
Gender: Female, Male
Target enrollment: 11 participants