Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

A Phase 1/2, First-in-Human, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of mRNA 3210 in Participants with Phenylketonuria

This research is an early-stage clinical trial looking into a new treatment for Phenylketonuria (PKU), a condition that affects how the body breaks down a substance called phenylalanine (Phe). The new medicine is called mRNA-3210 and it uses a technology similar to some vaccines, where a 'messenger' molecule tells your body how to make something it needs. The main goals of this study are to check if the new medicine is safe for people to take and to understand any side effects. Researchers will also be looking at how the body handles the medicine – how long it stays in your system and how it affects the levels of phenylalanine in your blood. This is important foundational work to see if this new approach could eventually help people with PKU manage their condition better.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Moderna Therapeutics Inc.

Enrolment target

Start

16 Jul 2024

Participants with Phenylketonuria

What is this study about?

Imagine your body is like a busy factory, and Phenylketonuria (PKU) is what happens when one of your factory machines isn't working quite right. Specifically, your body struggles to process a building block of protein called phenylalanine, which can then build up to harmful levels. This study is testing a new type of medicine that uses something called messenger RNA, or mRNA, to try and fix that 'broken machine'. You might have heard of mRNA because it's used in some modern vaccines.

In this study, the mRNA-3210 medicine aims to give your body instructions on how to make the missing or faulty 'machine' (an enzyme) that can process phenylalanine properly. By doing this, researchers hope to see if it can help reduce the levels of phenylalanine in the blood. This is an early, but very important, step to see if this new treatment approach is safe and has the potential to help people with PKU.

This is a 'first-in-human' study, which means it's one of the very first times this particular mRNA medicine is being given to people. Because it's so new, the main focus at this stage is to make sure it's safe and to understand how it behaves in the body. Researchers will be carefully checking for any side effects and seeing how the medicine affects blood phenylalanine levels. This will help them decide if this treatment shows enough promise to continue with further research.

Key takeaways

This is an early-stage study testing a new mRNA medicine for PKU.
The main focus is to check the medicine's safety and how it behaves in the body.
It aims to help the body process phenylalanine more effectively.
Participants must be adults with a diagnosis of Phenylketonuria.
You will be closely monitored for any side effects and changes in your health.

Who may be eligible?

To be able to join this study, you need to be an adult, aged 18 or older. The study welcomes both men and women.

Because this is an early study, there will be other specific health requirements to make sure it's safe for people to take part. These details aren't publicly available yet but would be discussed with you by the study team. Generally, you would need to have a diagnosis of Phenylketonuria (PKU).

If you're interested, the study doctors will go through your full medical history and current health to see if you meet all the criteria for taking part safely.

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 am 18 years old or older.
I have been diagnosed with Phenylketonuria (PKU).
I am comfortable with frequent medical check-ups and blood tests.
I understand that this is a very early study of a new medicine.

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive doses of the new medicine, mRNA-3210. Because it's an early-stage study, you would likely have regular visits to the clinic for check-ups. During these visits, the study team would monitor your health very closely. This would involve things like blood tests to check your phenylalanine levels and how the medicine is working, as well as general health checks.

To manage potential side effects or discomfort, you might also receive other common medicines like paracetamol or ibuprofen for pain, or antihistamines like cetirizine for allergies. The researchers will be collecting detailed information about your health throughout your time in the study. The total length of your participation would be explained fully by the study team, but early-stage trials often involve a period of receiving the medicine followed by a number of follow-up visits.

Potential risks and benefits

Taking part in a clinical trial can offer potential benefits, such as access to a new treatment before it's widely available, and helping advance medical knowledge for others with PKU. However, all new medicines carry potential risks and side effects, some of which may be unknown at this early stage. You might experience discomfort from procedures like blood tests, or side effects from the trial medicine itself, even if those side effects are mild. The study team will explain all known risks and monitor you closely. It's very important to remember that joining a study is always your choice, and you have the right to withdraw at any time for any reason, without it affecting your medical care.

Locations (3)

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

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Unverified
France
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Unverified
Spain
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Unverified
Italy

Common questions

What is Phenylketonuria (PKU)?

PKU is a rare genetic condition where your body can't properly break down a substance called phenylalanine, which is found in protein. This can lead to harmful build-up if not managed.

What is mRNA-3210 and how does it work?

mRNA-3210 is a new medicine that uses 'messenger RNA' (like a tiny instruction manual) to tell your body how to make a missing or faulty enzyme that usually processes phenylalanine.

What does 'first-in-human' mean?

It means this is one of the very first times this specific medicine is being given to people, so researchers are primarily focused on checking its safety and how the body handles it.

Are common medications allowed during the study?

Yes, common medications like paracetamol or ibuprofen for pain, or antihistamines for allergies, are listed as interventions, meaning they might be used to manage symptoms or mild side effects during the study.

What are the main goals of this study?

The main goals are to check how safe the new mRNA-3210 medicine is, how well people tolerate it, and how it moves through and affects the body, including phenylalanine levels.