In short
MZE782 is an investigational oral medication being studied for adults with phenylketonuria (PKU), a rare inherited condition where the body cannot properly process the amino acid phenylalanine. The EU Clinical Trials Information System lists one authorised Phase 2 trial of MZE782, planning to enrol 38 adults with PKU. The trial will measure how MZE782 affects phenylalanine levels in the blood over four weeks, as well as its safety and tolerability.
Key points
- MZE782 is an investigational oral drug for phenylketonuria (PKU) in adults.
- The only registered trial is a Phase 2 study that is currently authorised but not yet recruiting.
- The trial plans to enrol 38 adults with PKU who have blood phenylalanine levels above 600 μmol/L.
- Participants will take 480 mg MZE782 by mouth or a placebo tablet during a 4-week treatment period.
- The main goal is to measure the change in blood phenylalanine levels from the start of treatment to weeks 2, 3, and 4.
- Safety is also a key focus, including side effects, vital signs, laboratory tests, and heart rhythm recordings (ECGs).
What is MZE782?
MZE782 is an investigational oral medication being developed for adults with phenylketonuria (PKU), a rare inherited metabolic condition. In PKU, the body cannot properly break down the amino acid phenylalanine (Phe), which is found in many protein-containing foods. When phenylalanine builds up in the blood, it can affect brain function and overall health over time.
MZE782 is not yet an authorised medicine. The clinical trial registry data available for this substance lists no marketing authorisation, and the only registered study is still in the authorised stage, meaning it has received regulatory approval to begin but has not yet started recruiting participants. No trade names or other development codes for MZE782 appear in the available registry data.
How does MZE782 work?
The registry record for the Phase 2 trial states that the study aims to characterise the effect of MZE782 on plasma phenylalanine concentrations over a four-week treatment period in adults with PKU. However, the available trial data does not describe the molecular target or the exact mechanism by which MZE782 is expected to lower phenylalanine levels. No supplementary sources providing details on its mechanism of action were included in the data for this page, so the precise way MZE782 works in the body is not described here.
Which conditions is it studied for?
- Phenylketonuria (PKU) — a rare inherited condition in which the body cannot process the amino acid phenylalanine properly, leading to high levels in the blood that can harm the brain if untreated. The Phase 2 trial is specifically for adults with PKU whose baseline blood phenylalanine levels are above 600 μmol/L.
How is MZE782 given?
In the registered Phase 2 trial, MZE782 is given as a tablet for oral use at a dose of 480 mg. The trial also includes a matching placebo tablet, described in the registry as MZE782 F2 Placebo Tablets, which is used as the comparison arm. No other pharmaceutical forms or routes of administration for MZE782 are recorded in the available trial data.
What do the clinical trials show?
The EU Clinical Trials Information System lists one trial for MZE782: a Phase 2, interventional study in adults with phenylketonuria. The trial is currently authorised, meaning it has received regulatory approval to proceed but has not yet started recruiting participants. Because no results are available yet, the trial does not show any efficacy or safety findings at this stage.
The planned enrolment is 38 participants. The study is divided into cohorts: Cohorts 1A and 1B are the primary cohorts, and Cohort 2 is an additional cohort. Participants in all cohorts must be adults with PKU who have baseline plasma phenylalanine levels above 600 μmol/L.
The trial has two main goals. The first is to measure the absolute change in plasma phenylalanine levels from the start of treatment to the average of levels measured at weeks 2, 3, and 4, analysed separately for Cohorts 1A and 1B. The second is to assess safety and tolerability across all cohorts, based on the number of adverse events (side effects), changes in vital signs, clinical laboratory test results, and 12-lead electrocardiograms (ECGs), which record the electrical activity of the heart.
The study uses a double-blind, placebo-controlled (DBPC) design during the four-week treatment period, meaning neither the participants nor the researchers know who is receiving MZE782 and who is receiving the placebo until the study is completed.
What side effects are known?
No side effects of MZE782 are described in the available data. The Phase 2 trial is designed to collect information on safety and tolerability, including the incidence of adverse events, changes in vital signs, laboratory assessments, and ECG findings, but no results have been reported yet. Because MZE782 is still investigational and the trial has not started, the side effect profile of this substance is not yet known.