The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).
Eligibility
Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
Key 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
Key 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.
Primary outcome measure(s)
Safety and tolerability based on incidence of adverse events (AEs) — 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 — Baseline and Weeks 2, 3, and 4
Trial sites (6)
Facility
City
Region
Status
UCSF Benioff Children's Hospital Oakland
Oakland
California
Recruiting
Stanford University Clinical and Translational Research Unit
Palo Alto
California
Recruiting
Indiana University School of Medicine
Indianapolis
Indiana
Recruiting
University of Kentucky Medical Center
Lexington
Kentucky
Recruiting
Clinic for Special Children
Gordonville
Pennsylvania
Recruiting
University of Tennessee Health Science Center Le Bonheur Children's Hospital
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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