RecruitingPHASE2INTERVENTIONAL

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

The AGLOW study is a research project focusing on a new medicine, VX-407, for adults with Autosomal Dominant Polycystic Kidney Disease (ADPKD). This condition causes fluid-filled sacs (cysts) to grow in the kidneys, which can lead to kidney damage over time. The main goal of this study is to find out if VX-407 can slow down the growth of these cysts by measuring changes in kidney size. Researchers will also be checking if the medicine is safe to use, if people can tolerate it well, and how the body processes it. This early-stage study is an important step in understanding if VX-407 could be a future treatment option for ADPKD, specifically for patients with certain genetic features.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Vertex Pharmaceuticals Incorporated

Enrolment target

Start

19 Nov 2025

Estimated completion

31 Dec 2027

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

What is this study about?

This study, named AGLOW, is investigating a new medication called VX-407 for people living with Autosomal Dominant Polycystic Kidney Disease (ADPKD). ADPKD is a genetic condition where fluid-filled sacs, known as cysts, develop and multiply in the kidneys. Over time, these cysts can enlarge the kidneys and reduce their ability to function properly, potentially leading to kidney failure.

The main aim of this research is to see how VX-407 affects the size of the kidneys, as increased kidney size is often a sign of ADPKD progressing. Researchers will be measuring your kidney volume (adjusted for your height) to see if the medicine can slow down cyst growth. They also want to understand if VX-407 is safe for patients to use, if it causes any side effects, and how the body absorbs, distributes, and removes the medicine (what scientists call 'pharmacokinetics').

This is an early-stage study (Phase 2a), which means it's one of the first times this specific medicine is being tested in a larger group of patients to assess its effectiveness and safety. The insights gained from AGLOW will be crucial in deciding if VX-407 has the potential to become a treatment for ADPKD, particularly for individuals who have specific genetic changes in their PKD1 gene.

Key takeaways

This study is testing a new medicine (VX-407) for ADPKD.
It aims to see if the medicine can slow down kidney cyst growth.
Participation requires regular hospital visits and health monitoring.
You must be 18-65 years old and have a specific type of ADPKD.
Potential benefits and risks will be fully explained before you decide to join.
You can withdraw from the study at any time.

Who may be eligible?

To join this study, you must have been diagnosed with Autosomal Dominant Polycystic Kidney Disease (ADPKD) and be willing to attend all scheduled appointments and follow the study instructions. Your kidney cysts would also need to meet certain measurements seen on a special MRI scan (Mayo imaging classification 1B with a kidney size of at least 250 mL/m, 1C, 1D, or 1E).

Your kidneys also need to be working at a certain level. Specifically, your estimated kidney function (eGFR) must be 25 millilitres per minute per 1.73 square metre or higher. You must be between 18 and 65 years old, and the study is open to both men and women.

There are also some reasons why you wouldn't be able to join. For example, if you have another kidney condition not related to ADPKD, or if you've had a kidney or other organ transplant, or if you've had a kidney removed. People who are currently on dialysis or expect to start dialysis within the next year would also not be able to participate. The study team will review all your medical information to make sure it's safe and appropriate for you to join.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you have a diagnosis of ADPKD?
Are you between 18 and 65 years old?
Are you able to attend clinic visits regularly?
Has an MRI scan shown your kidney condition meets certain criteria (like Mayo 1B with a specific size, 1C, 1D, or 1E)?
Is your kidney function (eGFR) at least 25 mL/min/1.73 m² or higher?
Have you not had a kidney transplant or other major organ transplant?

Answer every question to see your result.

What does participation involve?

If you decide to take part in the AGLOW study, you will have regular visits to the clinic so the research team can monitor your health and the effects of the study medicine. These visits will involve various tests and assessments, including blood and urine samples, and possibly repeat MRI scans to measure your kidney size. You will be taking the study medicine, VX-407, as directed by the study team. You will also have follow-up appointments after you stop taking the medicine to ensure your health is still being monitored. The total duration of your involvement in the study will be explained in detail by the research team.

Potential risks and benefits

Taking part in any clinical study carries potential benefits and risks. For this study, a potential benefit could be that VX-407 might help slow down the progression of your ADPKD, although this is not guaranteed as it's a new medicine being tested. It's also possible you may not experience any direct benefits. Potential risks could include side effects from the medication or discomfort from study procedures like blood tests or MRI scans. The research team will explain all known or expected risks in detail. Remember, participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (42)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Alabama Kidney Research
Verified postcode
Alabaster, United States· Recruiting
UAB Nephrology Research at CH19
Verified postcode
Birmingham, United States· Recruiting
Kaiser Permanente Los Angeles Medical Center
Verified postcode
Los Angeles, United States· Recruiting
UCSF Clinical Research Center at Parnassus
Verified postcode
San Francisco, United States· Recruiting
Nephrology & Hypertension Associates, PC
Verified postcode
Middlebury, United States· Recruiting
Yale University - Yale Nephrology Clinical Research Clinic
Verified postcode
New Haven, United States· Recruiting
Mayo Clinic - Jacksonville
Verified postcode
Jacksonville, United States· Recruiting
Emory School of Medicine - Renal Division
Verified postcode
Atlanta, United States· Recruiting
Renal Associates
Verified postcode
Columbus, United States· Recruiting
UChicago Medicine - Duchossois Center for Advanced Medicine - Nephrology
Verified postcode
Chicago, United States· Recruiting
University of Iowa Health Care Medical Center- Nephrology
Verified postcode
Iowa City, United States· Recruiting
Clinical & Translational Science Unit (CTSU) - Nephrology
Verified postcode
Kansas City, United States· Recruiting

Common questions

What is ADPKD?

ADPKD stands for Autosomal Dominant Polycystic Kidney Disease. It's a genetic condition where fluid-filled sacs called cysts grow in your kidneys, which can affect their function over time.

What is VX-407?

VX-407 is a new medicine being studied. This research project is trying to find out if it's safe and effective for treating ADPKD.

Will I definitely get the new medicine?

In some studies, participants are randomly assigned to receive the study medicine or a placebo (a dummy medicine). The researchers will explain exactly what this means for you.

Will I have to pay to join this study?

No, you will not have to pay to participate in the study, and study-related medical care and the study medicine will be provided free of charge. Travel expenses may also be reimbursed.

What if I change my mind about participating?

You have the right to leave the study at any time, for any reason, without having to explain why. Your decision will not affect your ongoing medical care.

How to find out more

Medical Information

medicalinfo@vrtx.com 617-341-6777 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.