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

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. To evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV) in patients with a subset of variants of the PKD1 gene

CT.gov Identifier
NCT07161037
EudraCT Identifier
N/A
CTIS:
2024-517393-13-00
Sponsor
Vertex Pharmaceuticals
Collaborator
N/A
Study Contact Information
+1 617 341 6100
Recruiting
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Study Details

Diseases
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Study Drug
VX-407
Genes
PKD1
Study Dates
Sep 2025 - Jul 2027
Sex
Female & Male
Age
18 - 65 Years
Inclusion and Exclusion Criteria
Inclusion Criteria:
  • Key
  • * 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.

Protocol 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. To evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV) in patients with a subset of variants of the PKD1 gene

Study Locations

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