NCT06061614 Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
| NCT ID | NCT06061614 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | The First Affiliated Hospital of Bengbu Medical University |
| Condition | Phenylketonurias |
| Study Type | INTERVENTIONAL |
| Enrollment | 15 participants |
| Start Date | 2023-03-30 |
| Primary Completion | 2028-12-30 |
Trial Parameters
Eligibility Fast-Check
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Brief Summary
This is a single-center, open-label, non-randomized, dose escalation study to evaluate the safety, tolerability and efficacy of NGGT002 in adult Phenylketonuria (PKU) subjects. All subjects will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
Eligibility Criteria
Inclusion Criteria: * Voluntarily sign informed consent form; * Male and female subjects with diagnosis of PKU caused by confirmed phenylalanine hydroxylase(PAH) mutation according to the "Clinical Practice Guidelines for Phenylketonuria, 2020 Edition"; * Age ≥ 18 years; * Blood phenylalanine (Phe) concentration ≥ 600 μmol/L at least once within 2 years prior to screening, with one measurement confirmed within six months of enrollment; * Subjects are able to maintain their baseline diet throughout the study (regardless of dietary phenylalanine restriction), and willingness to follow the instruction of investigators to manage the diet for the duration of the trial; * Subjects are required to obtain approval from the investigator prior to the use of any concomitant medications during the study period; * Willingness and capable per Investigator opinion to comply with study procedures and requirements; * Female participants of childbearing potential must have abstained from unprotected sex