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

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-517393-13-00

Acronym

VX24-407-101

Enrollment

Registered

2025-11-28

Start date

2025-12-02

Completion date

Unknown

Last updated

2025-12-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Brief summary

Proportion of subjects with percent change from baseline in htTKV ≤0 on MRI over time

Detailed 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

Interventions

DRUGVX-407 tablet

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Proportion of subjects with percent change from baseline in htTKV ≤0 on MRI over time	—

Secondary

Measure	Time frame
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	—

Countries

Belgium, France, Germany, Italy, Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026