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Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants

Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants

Recruiting
18-65 years
All
Phase 2

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Overview

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

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 impact the natural history of ADPKD
  • History of solid organ or bone marrow transplantation or nephrectomy
  • Ongoing renal replacement therapy or planning to start renal replacement therapy within 12 months of screening

Other protocol defined Inclusion/Exclusion criteria will apply.

Study details
    Autosomal Dominant Polycystic Kidney Disease (ADPKD)

NCT07161037

Vertex Pharmaceuticals Incorporated

31 October 2025

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