Health TrialsbyHealthPanel

A Study of MZE782 in Adults With PKU

ConditionPhenylketonuria (PKU)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorMaze Therapeutics

About this trial

The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Males and females 18 to 75 years old

Confirmed diagnosis of PKU

Willing and able to keep diet and protein intake consistent during the study

For some study groups, is willing and able to stop certain PKU medications before starting study treatment

Disqualifiers

Is pregnant, breastfeeding, or planning to become pregnant or conceive a child during the study

Medical conditions or abnormal test results that could make participation unsafe or make it difficult to complete study procedures or interpret study results

Has recently received another investigational drug

Is unable to swallow tablets or tolerate oral medication

Trial design

Design model

Parallel

Treatments tested in this trial

  • MZE782

    Drug

    Tablet, Low Dose

  • Placebo

    Other intervention

    Tablet

  • MZE782

    Drug

    Tablet, High Dose

Treatment groups

38 Participants
are divided into 6 treatment groups

6

Treatment groups

See each treatment group below.

Group A: MZE782 Low Dose (Cohort 1A)Experimental treatment 1 intervention
Group B: Placebo (Cohort 1A)Placebo comparator 1 intervention
Group C: MZE782 High Dose (Cohort 1B)Experimental treatment 1 intervention
Group D: Placebo (Cohort 1B)Placebo comparator 1 intervention
Group E: MZE782 High Dose (Cohort 2)Experimental treatment 1 intervention
Group F: Placebo (Cohort 2)Placebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Safety and tolerability based on incidence of adverse events (AEs)

Time frame
Week 0 to Week 17
2

For Cohorts 1A and 1B: Absolute change in plasma Phe levels from Baseline to the mean of plasma Phe levels

Time frame
Baseline and Weeks 2, 3, and 4

Secondary outcomes

1

For Cohorts 1A and 1B, analyzed separately: Geometric mean plasma concentrations of MZE782

Time frame
Weeks 1, 2, 3, 4, 8, and 13
2

For Cohorts 1A and 1B, analyzed separately: Absolute and Percent change in plasma Phe levels from Baseline to the mean of plasma Phe levels

Time frame
Baseline up to Week 13
3

For Cohorts 1A and 1B, analyzed separately: Proportion of participants with mean plasma Phe levels ≤600 μMol/L, ≤360 μMol/L, and ≤120 μMol/L

Time frame
Baseline and Weeks 2, 3, 4, 8, 10, and 13

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Maze Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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