Recruiting NCT07694440
A Study of MZE782 in Adults With PKU
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: MZE782, Placebo, MZE782.
- Who it may be relevant to
- Registry conditions: Phenylketonuria (PKU). Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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)
Overview
The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).
Interventions
- Drug MZE782
Tablet, Low Dose - Other Placebo
Tablet - Drug MZE782
Tablet, High Dose
Primary outcome measures
- Safety and tolerability based on incidence of adverse events (AEs) [Time frame: Week 0 to Week 17]
- 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 outcome measures (3)
- For Cohorts 1A and 1B, analyzed separately: Geometric mean plasma concentrations of MZE782 [Time frame: Weeks 1, 2, 3, 4, 8, and 13]
- 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]
- 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]
Eligibility criteria
Inclusion criteria
- Males and females 18 to 75 years old
- Clinical 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
- For some study groups, may need to meet additional blood phenylalanine level or background-treatment requirements
- Meets minimum blood phenylalanine level requirements for study group
Exclusion criteria
- Is pregnant, breastfeeding, or planning to become pregnant or conceive a child during the study or within 90 days after the last dose
- 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
Other inclusion/exclusion criteria defined in protocol apply.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- Indiana University School of Medicine — Indianapolis
- Clinic for Special Children — Gordonville
Identifiers
NCT: NCT07694440 · MZE782-201