A Phase 2, Randomized, Double-blind, Placebo-controlled, Dose-ranging Study to Evaluate the Safety, Tolerability, and Efficacy of MZE782 in Adults with Phenylketonuria (PKU)
Phenylketonuria (PKU) is a lifelong condition where the body cannot properly break down phenylalanine (Phe), a natural substance found in protein-rich foods. Without treatment, Phe builds up in the blood and can cause health problems. This Phase 2 trial is looking at an experimental tablet called MZE782 in adults with PKU. Researchers want to find out how safe the medicine is, how well the body tolerates it, and whether it successfully lowers blood Phe levels. Participants will be placed into groups to test different doses of MZE782 or a placebo (a dummy pill with no active medicine). Doctors will closely track participants' health through regular blood tests, heart checks, and physical exams over several weeks to see how effective the treatment is and if it causes any unwanted side effects.
At a glance
What is this study about?
Phenylketonuria, often shortened to PKU, is a rare genetic condition that affects how the body processes protein. People with PKU lack enough of an enzyme needed to break down phenylalanine, or Phe. Phe is an amino acid found in everyday foods like meat, dairy, eggs, and nuts. When Phe levels climb too high in the blood, it can affect concentration, mood, and long-term brain health. Currently, most adults with PKU must follow very strict, low-protein diets and drink special medical formulas to keep their levels in check.
This research study is investigating a new tablet called MZE782. Scientists hope that this medicine can help lower the amount of Phe in the blood, potentially offering an easier way to manage the condition. Because this is a Phase 2 trial, doctors are trying to figure out the right dose to use and check whether the treatment works as intended while remaining safe.
To find out if MZE782 works, researchers will compare it against a placebo. A placebo looks identical to the real tablet but contains no active medicine. Neither the study participants nor the doctors will know who is taking the medicine and who is taking the placebo during the study period. Comparing both groups helps scientists prove whether any changes in blood Phe levels are truly caused by MZE782 rather than chance.
Key takeaways
- The trial tests an experimental tablet called MZE782 for adults living with PKU.
- The study aims to check the safety of the drug and see if it lowers blood phenylalanine (Phe) levels.
- Participants will receive either the study medicine at different doses or a placebo tablet.
- You will need to attend regular clinic visits for blood tests, heart checks, and physical assessments over several weeks.
- Taking part is completely voluntary, and you can withdraw at any time.
Who may be eligible?
This clinical trial is open to adults aged 18 and older who have been diagnosed with phenylketonuria (PKU). Both men and women can take part.
Because the study focuses on adult care, anyone under 18 years of age is not able to join. As with most clinical trials, there are additional health and medical checks that the study team will carry out to make sure it is safe for you to take part.
If you are interested, the trial doctor will review your medical history, any medicines you currently take, and your usual blood Phe levels to confirm whether this study is suitable 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 am aged 18 or older.
- I have a confirmed diagnosis of phenylketonuria (PKU).
- I am willing to take daily study tablets as instructed.
- I am able to attend regular hospital or clinic appointments for blood tests and heart checks.
- I am happy to follow the trial guidelines and keep my usual diet stable during the study.
What does participation involve?
Taking part in this study involves taking a tablet by mouth for several weeks. You will be randomly assigned to receive either a specific dose of the study medicine (MZE782) or a dummy pill (placebo).
Throughout the study, you will need to attend regular clinic visits, especially during the first month and at key points up to around 13 weeks. At these visits, the study team will take blood samples to measure your Phe levels and see how much medicine is in your body. They will also carry out routine health checks, such as checking your blood pressure, performing physical exams, and taking electrocardiograms (ECGs) to monitor your heart rhythm.
You will be asked to report any symptoms or side effects you notice. The study team will follow your progress closely to ensure your safety from the start of treatment through to the final follow-up visit.
Potential risks and benefits
Locations (3)
- —UnverifiedGermany
- —UnverifiedFrance
- —UnverifiedNetherlands
Common questions
What is the main goal of this clinical trial?
The main goal is to see if the new medicine MZE782 safely and effectively reduces phenylalanine (Phe) levels in the blood of adults with PKU.
What is a placebo and why is it used?
A placebo is a dummy pill that looks just like the real medicine but has no active ingredients. It helps scientists clearly see if changes in health are genuinely caused by the study drug.
Will I know if I am taking the real medicine or the placebo?
No. This is a 'double-blind' study, meaning neither you nor your study doctor will know which tablet you receive until the trial is finished.
Do I have to change my usual PKU diet to take part?
The study team will give you specific guidelines about your diet. In general, it is important to keep your protein intake as stable as possible during the trial so blood results remain accurate.
Can I leave the study if I change my mind?
Yes. You have the right to withdraw from the trial at any point, for any reason, and your normal healthcare will continue as usual.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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