Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
ConditionPhenylketonurias
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorThe First Affiliated Hospital of Bengbu Medical University
About this trial
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
Qualifiers
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;
Disqualifiers
Anti-AAV8 neutralizing antibody>1:10
Prior gene therapy
Positive hepatitis B virus surface antigen, hepatitis C virus antibody, anti-human immunodeficiency virus antibody or treponema pallidum-specific antibody
Hepatic function abnormal: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 1.5 × ULN; alkaline phosphatase (ALP) > 1.5 × ULN; total bilirubin (TBil) > 1.5 × ULN; international normalized ratio (INR) > 1.3
Trial design
Treatments tested in this trial
- NGGT002 Injection
Treatment groups
15 Participants
are divided into 5 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov