RecruitingPHASE1INTERVENTIONAL

A Study to Evaluate the Safety, Tolerability, and Effects on Blood and Urine Markers of Single Ascending Dose of GSK4771261 in Healthy Participants and Participants With Autosomal Dominant Polycystic Kidney Disease

This study is testing a new medication called GSK4771261. It's a 'Phase 1' study, which means it's one of the first steps in checking if a new drug is safe and how it works in people. No one, not even the doctors or participants, will know who is getting the new drug or a dummy pill. The study has two main parts. Part A involves healthy volunteers to see if the new drug is safe and how their bodies react to it. Part B is similar but focuses on people who have a type of kidney disease called autosomal dominant polycystic kidney disease (ADPKD). Researchers want to see if the drug affects their blood and urine markers.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

GlaxoSmithKline

Enrolment target

Start

11 Dec 2024

Estimated completion

04 Sep 2026

Kidney Disease

What is this study about?

This study is trying to understand more about a new medication called GSK4771261. Think of it as a very early stage of testing for a new drug. The main goals are to check if it's safe for people to take, how much of it stays in the body, and what effects it has on various measurements in your blood and urine.

The study is split into two groups. The first group, called Part A, involves healthy volunteers. This helps researchers understand the basic safety of the drug and how it works in bodies that aren't already affected by illness. The second group, Part B, will include people who have a specific inherited kidney condition called autosomal dominant polycystic kidney disease (ADPKD). By studying people with ADPKD, the researchers hope to learn if this new drug could potentially help manage or treat this condition.

It's important to know that this is a 'blinded' study. This means neither you, nor the study doctors, will know whether you are receiving the new drug or a 'placebo' – which looks identical but contains no active medicine. This helps make sure the study results are fair and unbiased. The findings from this study will help decide if this new drug should be tested further in larger studies.

Key takeaways

This study is testing a brand-new drug, GSK4771261, for safety and its effects.
It involves both healthy people (Part A) and those with ADPKD (Part B).
No one will know if they are receiving the drug or a 'dummy' pill.
The study checks blood and urine changes as well as general safety.
It's an early-stage 'Phase 1' study for potential future ADPKD treatment.

Who may be eligible?

To join this study, you generally need to be a man or a woman aged between 25 and 65 years old. You also need to weigh at least 45 kilograms (about 7 stone 1 pound) and have a healthy body mass index (BMI) between 19.5 and 32. All participants must be able to understand the study and agree to take part by signing a consent form.

For Part A, you must be a healthy adult with no major ongoing health problems, as assessed by the study doctor. For women, you must have gone through menopause, had a hysterectomy or fallopian tubes tied, or have other reasons why you can't have children.

If you have Autosomal Dominant Polycystic Kidney Disease (ADPKD) and are interested in Part B, you must have a confirmed diagnosis of this condition. While you might have some health issues related to ADPKD, you should otherwise be in good general health. Your kidney function, measured by something called eGFR, needs to be reasonably good (at least 45 mL/min/1.73m²), and your doctor shouldn't expect it to get much worse quickly. You'll also need to have certain types of ADPKD based on imaging scans and a confirmed genetic fault related to the condition.

Quick self-check

Are you between 25 and 65 years old?
Do you weigh at least 45 kilograms (about 7 stone 1 pound)?
Is your Body Mass Index (BMI) between 19.5 and 32?
For women, are you unable to have children (e.g., post-menopause, sterilised)?
For Part B, do you have a confirmed diagnosis of ADPKD?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you will be given either the new drug, GSK4771261, or a 'dummy' pill (placebo) that looks exactly the same. No one involved will know which you are receiving. You will need to come for several visits to the study clinic where doctors will check your health, take blood and urine samples, and monitor how your body is reacting to the treatment. They'll also perform other tests like physical exams and heart checks. The study length isn't specified, but Phase 1 studies often involve regular visits over weeks or months, followed by a period where researchers continue to check on your health. You will be told the exact schedule of visits and tests during the consent process.

Potential risks and benefits

Participating in a clinical trial like this could offer potential benefits, such as contributing to scientific understanding about ADPKD and potentially gaining early access to a new treatment, though there's no guarantee the treatment will directly benefit you. However, there are also potential risks involved, mainly from the new drug itself, which might cause side effects that we don't fully know about yet. You might also experience discomfort from blood tests and other procedures. It's really important to remember that you can stop participating in the study at any time, for any reason, without it affecting your usual medical care.

Locations (14)

GSK Investigational Site
Brussels, Belgium· Recruiting
GSK Investigational Site
Leuven, Belgium· Recruiting
GSK Investigational Site
London, Canada· Recruiting
GSK Investigational Site
Montreal, Canada· Recruiting
GSK Investigational Site
Brest, France· Recruiting
GSK Investigational Site
Cologne, Germany· Recruiting
GSK Investigational Site
Barcelona, Spain· Recruiting
GSK Investigational Site
Barcelona, Spain· Recruiting
GSK Investigational Site
Madrid, Spain· Recruiting
GSK Investigational Site
Madrid, Spain· Recruiting
GSK Investigational Site
Cambridge, United Kingdom· Recruiting
GSK Investigational Site
Exeter, United Kingdom· Recruiting

+2 more sites — see the official record for the full list.

Common questions

What is Autosomal Dominant Polycystic Kidney Disease (ADPKD)?

ADPKD is an inherited kidney condition where cysts (fluid-filled sacs) grow in the kidneys, which can cause them to become very large and affect how well they work.

What is a 'Phase 1' study?

Phase 1 is the earliest stage of human testing for a new drug. It mainly checks if the drug is safe, what dose is best, and how the body handles it.

What does 'blinded' mean in this study?

Being 'blinded' means neither you nor the study doctors will know if you are receiving the new drug or a dummy pill (placebo). This helps make the study fair.

Will I know if the new drug is working for me?

Because this is an early-stage study and it's 'blinded,' you won't know if you're getting the active drug or a placebo during the study. The main focus is on safety and how your body reacts.

Can I stop participating in the study if I change my mind?

Yes, absolutely. You are free to withdraw from the study at any time, for any reason, without it affecting your normal medical care.

How to find out more

US GSK Clinical Trials Call Center

GSKClinicalSupportHD@gsk.com 877-379-3718 Official trial record

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