Phase 2a Study of VX-407 in Participants with ADPKD Who Have a Subset of PKD1 Gene Variants
Research type
Research Study
Full title
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
IRAS ID
1012156
Contact name
Devi Smart
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Research summary
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a genetic disease which causes cysts (fluid-filled sacs) to grow in the kidneys. As the cysts grow, the kidneys may stop working properly and can lead to kidney failure.
Right now, there are no treatments that fix the root cause of ADPKD. In most people with this condition (about 80%), it is caused by a change in a gene called PKD1. This gene normally tells the body to make a protein called polycystin 1 (PC1). If PKD1 is not working right, PC1 doesn't work either. VX-407 is an investigational drug that may help fix the faulty PC1 protein.
This study is called a Phase 2a, open-label, single-arm study. That means everyone in the study will take the same drug (VX-407), and they will all know they are getting it. The goal is to see if the drug is safe, if it's easy to take, and how it affects people with ADPKD who have the PKD1 gene.
The study includes a screening period that will last up to 28 days, a 52-week study treatment period with 11 visits, and a safety follow-up visit that will take place 14 days after last dose of Study Drug. Participants will take the study drug by mouth, twice a day. Study activities include physical examinations, electrocardiograms (ECGs), abdominal magnetic resonance imaging (MRIs), blood and urine tests.
About 24 participants, male and female, ages 18 to 65 years old, will participate in this study around the world. The sponsor of this trial is Vertex Pharmaceuticals Inc.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
25/SC/0227
Date of REC Opinion
2 Sep 2025
REC opinion
Further Information Favourable Opinion