Status:
RECRUITING
Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
Lead Sponsor:
The First Affiliated Hospital of Bengbu Medical University
Conditions:
Phenylketonurias
Eligibility:
All Genders
18+ years
Phase:
EARLY_PHASE1
Brief Summary
This investigator initiated trial is an open-label, dose escalation study to evaluate the safety, tolerability and efficacy of NGGT002, an AAV derived investigational gene therapy product expressing h...
Detailed Description
This study will evaluate the safety and efficacy of NGGT002 gene therapy with three dose cohorts in adult subjects with diagnosis of PKU characterized by PAH deficiency with confirmed PAH gene mutatio...
Eligibility Criteria
Inclusion
- Voluntarily sign informed consent form;
- Male and female subjects with diagnosis of PKU caused by confirmed PAH mutation as per the "Clinical Practice Guidelines for Phenylketonuria 2020";
- Age ≥ 18 years;
- Phe concentration ≥ 600 μmol/L at screening and ≥ 600 μmol/L at least once within 2 years prior to screening;
- Subjects who are willing and able to maintain their baseline diet throughout the study, regardless of phenylalanine restriction, except at the investigator's request;
Exclusion
- Presence of anti-AAV8 neutralizing antibody
- 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
Key Trial Info
Start Date :
March 30 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 30 2028
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06061614
Start Date
March 30 2023
End Date
December 30 2028
Last Update
February 7 2024
Active Locations (1)
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1
The First Affiliated Hospital of Bengbu Medical College
Bengbu, Anhui, China, 233000