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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

This trial does not accept healthy volunteers

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

Design model

Sequential

Treatments tested in this trial

  • NGGT002 Injection

    Genetic

    Dose escalation will proceed sequentially from Dose Level 1 to Dose Level 5 in a single-dose administration of NGGT002.

Treatment groups

15 Participants
are divided into 5 treatment groups
Group A: Dose level 1Experimental treatment 1 intervention
Group B: Dose level 2Experimental treatment 1 intervention
Group C: Dose level 3Experimental treatment 1 intervention
Group D: Dose level 4Experimental treatment 1 intervention
Group E: Dose level 5Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)

Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) from baseline to week 52 (W52)

Time frame
Week 52

Secondary outcomes

1

Plasma Phe levels

Changes from baseline in plasma Phe levels

Time frame
Baseline Week 12, Week 28, Week 52
2

Nature protein intake

Changes from baseline in nature protein intake (g/kg body weight \[BW\]/day)

Time frame
Baseline, Week 12, Week 28, and Week 52
3

Food Phe intake

Changes from baseline in food Phe intake (mg/kg body weight \[BW\]/day)

Time frame
Baseline, Week 12, Week 28, and Week 52

Other outcomes

Sponsors and contacts

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

The First Affiliated Hospital of Bengbu Medical University

Lead sponsor

Source ClinicalTrials.gov
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