VX-407 for Polycystic Kidney Disease
(AGLOW Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called VX-407, a potential drug for polycystic kidney disease, where clusters of cysts develop in the kidneys. The goal is to assess how VX-407 affects kidney size, safety, and drug processing in the body. Individuals diagnosed with a specific type of polycystic kidney disease who can undergo certain study procedures may qualify. Eligibility includes having larger kidneys as seen on an MRI and a certain level of kidney function. Participants will receive the treatment for up to 52 weeks to evaluate its effectiveness and safety. As a Phase 2 trial, this research focuses on measuring the treatment's efficacy in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that VX-407 is likely to be safe for humans?
Research shows that VX-407 has undergone safety testing in healthy individuals. In these studies, most participants tolerated VX-407 well, experiencing no serious side effects. Since this study is in Phase 2, VX-407 has already passed initial safety tests in earlier trials. This phase focuses more on the treatment's safety and effectiveness for people with polycystic kidney disease (PKD). The treatment aims to address a specific genetic issue related to PKD. Although more information is needed, results so far are promising for safety.12345
Why do researchers think this study treatment might be promising?
Most treatments for polycystic kidney disease, like tolvaptan, aim to slow kidney function decline by regulating fluid balance. But VX-407 works differently. Researchers are excited about VX-407 because it targets the underlying genetic mutations responsible for cyst formation, potentially offering a more precise approach. This innovative mechanism could lead to fewer side effects and improved outcomes compared to current therapies, making it a promising option for patients.
What evidence suggests that VX-407 might be an effective treatment for Polycystic Kidney Disease?
Research has shown that VX-407, the investigational treatment in this trial, could be a promising option for autosomal dominant polycystic kidney disease (ADPKD). It addresses a protein problem in kidney cells, potentially reducing cyst growth. This treatment aims to slow the increase in kidney size, a common issue in ADPKD. Early results suggest that this small molecule effectively targets the disease's root cause in patients with certain genetic types. Although human trial data remains limited, the treatment's design shows potential to address the condition's main problems.12367
Are You a Good Fit for This Trial?
This trial is for people with a specific type of Polycystic Kidney Disease (ADPKD) who have certain PKD1 gene variants. Participants must be able to follow the study schedule, have an ADPKD diagnosis confirmed by MRI, and a kidney function test result (eGFR) of at least 25 mL/min/1.73 m².Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive VX-407 for up to 52 weeks to evaluate its effect on height-adjusted total kidney volume, safety, tolerability, and pharmacokinetics
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- VX-407
Find a Clinic Near You
Who Is Running the Clinical Trial?
Vertex Pharmaceuticals Incorporated
Lead Sponsor
Dr. David Altshuler
Vertex Pharmaceuticals Incorporated
Chief Medical Officer since 2020
MD, PhD
Dr. Reshma Kewalramani
Vertex Pharmaceuticals Incorporated
Chief Executive Officer since 2020
MD, trained in internal medicine and nephrology