Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Double-blind, Placebo-controlled, Randomized Withdrawal Study to Evaluate the Efficacy and Safety of SYNB1934 in Patients with PKU (SYNPHENY-3)

This research study is looking at a new medicine called SYNB1934 for individuals with Phenylketonuria (PKU). PKU is a condition where the body can't properly break down a substance called phenylalanine (Phe), which can then build up in the blood. The main goals of this study are to find out how effective SYNB1934 is at reducing Phe levels in the blood and to monitor any side effects to make sure it's safe. Participants will be given either the new medicine or a dummy medicine (placebo) to compare the results fairly. This is a very important step in seeing if SYNB1934 could become a helpful new treatment option for people living with PKU.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Synlogic Inc.

Enrolment target

Start

16 Jan 2024

Phenylketonuria

What is this study about?

This research study is for people who have a condition called Phenylketonuria, often shortened to PKU. If you have PKU, your body has trouble breaking down a substance called phenylalanine, or Phe for short. Phe is found in many foods, especially protein-rich foods, and if it builds up too much in your blood, it can cause problems. Currently, managing PKU often involves a strict diet.

This study is testing a new medicine called SYNB1934. The main purpose is to see how well this medicine can help lower the amount of Phe in the blood. Researchers also want to understand if the medicine is safe and what side effects it might cause. By carefully comparing people who receive the new medicine with those who receive a dummy medicine (a placebo), the study aims to get clear answers.

This type of study, called a 'Phase 3' study, is a big and important step. It helps confirm if a new medicine works effectively and safely before it can be made available more widely. The results from this study will help doctors and patients understand if SYNB1934 could be a useful new way to manage PKU.

Key takeaways

This study is testing a new medicine (SYNB1934) for Phenylketonuria (PKU).
Its main goals are to see how well it lowers Phe levels and if it's safe.
Participants will receive either the new medicine or a dummy medicine (placebo).
It's a 'Phase 3' study, meaning it's a big step towards potential new treatments.
Participation involves regular visits and health checks.

Who may be eligible?

This study is designed for adults aged 18 years and older who have been diagnosed with Phenylketonuria (PKU). Both men and women are welcome to take part in the study.

Before you can join, the study team will need to check some other things about your medical history and current health to make sure the study is a good fit for you and that it would be safe for you to participate. This helps ensure everyone in the study meets the specific criteria needed for the research.

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.

Are you 18 years old or older?
Have you been diagnosed with Phenylketonuria (PKU)?
Are you able to attend regular clinic visits?
Are you willing to potentially receive either the study medicine or a placebo?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you might be given the new medicine (SYNB1934), or a placebo, which looks like the medicine but has no active ingredient. You'll switch between these during the study. You will have regular visits to the clinic so doctors can monitor your blood phenylalanine (Phe) levels, check your general health through blood tests and vital signs (like blood pressure), and ask about any side effects you might be experiencing. The study duration will vary depending on which part you are in, but it will involve several visits to the study site and careful follow-up over a period of time.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit: you could receive a new treatment that might help lower your Phe levels, which could improve your health. However, there are also potential risks, such as experiencing side effects from the study medicine or discomfort from blood tests. We don't yet know if the new medicine will be effective or which side effects it might cause. Remember, participating is entirely your choice, and you are free to withdraw from the study at any time without affecting your usual 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
Denmark

Common questions

What is Phenylketonuria (PKU)?

PKU is a genetic condition where your body can't properly break down a substance called phenylalanine (Phe), which is found in many foods. This can lead to high levels of Phe in the blood.

What is SYNB1934?

SYNB1934 is a new medicine being tested to help people with PKU lower their phenylalanine (Phe) levels in the blood.

What does 'double-blind' mean?

It means neither you nor your study doctor will know if you are receiving the active medicine (SYNB1934) or a dummy medicine (placebo). This helps make the study results fair and unbiased.

What is a 'placebo'?

A placebo is a substance that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers compare the effects of the new medicine to not having any active treatment.

How long will the study last?

The study has different parts, and the exact length of your participation will be explained by the study team. It will involve several visits over a period of time.