AuthorisedHuman Pharmacology (Phase I)- OtherInterventional

Evaluation of AG-181 in Subjects 18 to 69 Years of Age With Phenylketonuria

This research is testing a new medicine called AG-181 in adults aged 18 to 69 who have a condition called phenylketonuria (PKU). PKU is a lifelong genetic condition where the body can't properly break down a substance found in many foods. This early study, known as a Phase 1 trial, is mainly focused on learning more about how AG-181 behaves in the human body. Researchers want to see how much of the medicine gets into the blood, how it's processed, and how quickly it leaves the body. This helps them understand if it's safe to use and how best to give it. It's a crucial first step in developing potential new treatments for PKU.

At a glance

Status

Authorised

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Agios Pharmaceuticals Inc.

Enrolment target

Start

03 Apr 2026

Phenylketonuria

What is this study about?

This study is designed to explore a new potential medicine, called AG-181, for individuals living with phenylketonuria (PKU). PKU is a condition people are born with where their body cannot properly process a part of protein called phenylalanine. If too much phenylalanine builds up, it can cause health problems. People with PKU usually manage their condition through a very strict diet.

This particular study is a 'Phase 1' trial, which means it's one of the first times this new medicine is being given to people. The main goal at this stage isn't to see if it cures PKU, but rather to understand how it works inside the body. Scientists want to learn things like how the medicine is absorbed, how it's broken down, and how it leaves the body. This information is really important for deciding if the medicine can be studied further in larger groups of people and for figuring out the right dose.

Studies like this are vital because they help researchers discover new ways to manage conditions like PKU. Even though it's an early study, the information gathered from volunteers is crucial for developing future treatments that could potentially improve the lives of people with PKU.

Key takeaways

This study is testing a new medicine (AG-181) for PKU.
It's an early-stage (Phase 1) study to understand how the medicine works in the body.
Adults aged 18 to 69 (and potentially older) with PKU can be considered.
Both men and women are eligible.
Participation involves medical checks and likely blood tests.
Your contribution could help develop future PKU treatments.

Who may be eligible?

This study is looking for adults who have been diagnosed with phenylketonuria (PKU). To be considered, you need to be at least 18 years old. There is no upper age limit specified, so adults over 69 could potentially take part.

Both men and women are welcome to participate in this research. The study team will need to check your medical history and current health carefully to make sure that taking part would be safe for you. They will have specific health checks and criteria that you would need to meet.

It's important to remember that this is a very early study. If you're interested, the research team will explain all the requirements in detail and answer any questions you have to help you decide if it's right for you.

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.

I have been diagnosed with phenylketonuria (PKU).
I am 18 years old or older.
I understand this is an early-stage study.
I am willing to follow study instructions and attend appointments.

Answer every question to see your result.

What does participation involve?

The detailed information about what taking part involves is not available in the brief summary. However, in a typical Phase 1 study like this, you would likely have several visits to the study clinic. These visits often include medical checks, blood tests to measure the medicine in your body, and possibly providing other samples like urine. You would be given the study medicine, AG-181, and carefully monitored to see how your body reacts. The total duration of the study and how many times you would need to visit would be explained fully by the research team if you are deemed eligible.

Potential risks and benefits

Participating in any research study carries potential benefits and risks. A potential benefit of this study is contributing to the development of new treatments for PKU, which could help future patients. However, as it's an early-stage study, the direct individual benefit to you might not be known, and there's no guarantee the medicine will help your condition. Potential risks could include side effects from the study medicine, discomfort from blood tests, or unforeseen reactions. You would be closely monitored. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

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

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Unverified
Poland

Common questions

What is phenylketonuria (PKU)?

PKU is a condition where your body can't properly break down a substance found in protein. It's usually managed with a special diet.

What is a Phase 1 study?

It's an early-stage study that mainly looks at how a new medicine works in the body and if it's safe, rather than if it cures a condition.

Will this medicine help my PKU?

In this early study, the main goal is to understand the medicine, not to see if it cures PKU. Any direct benefit to you might not be known yet.

Do I have to pay to join the study?

No, you should not have to pay to participate in a clinical trial. Study-related medical care is typically covered by the trial sponsor.

Can I leave the study if I change my mind?

Yes, you are free to leave the study at any time, for any reason, without it affecting your usual medical care.