Safety and Efficacy Study of NGGT002 in PKU Adult Subjects

Launched by THE FIRST AFFILIATED HOSPITAL OF BENGBU MEDICAL UNIVERSITY · Sep 25, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called NGGT002 to see if it is safe and effective for adults with Phenylketonuria (PKU), a genetic condition that affects how the body processes certain proteins. In this study, all participants will receive a single dose of NGGT002 and will be monitored for five years to track their health and how well the treatment works. The goal is to help people with PKU manage their condition better.

To participate in this trial, individuals must be at least 18 years old and have a confirmed diagnosis of PKU due to a specific genetic mutation. They should also have high levels of a substance called phenylalanine (Phe) in their blood. Participants need to be willing to continue following their usual diet throughout the study. It's important to note that individuals with certain prior health issues, like previous gene therapy or liver problems, may not be eligible. If you or a loved one is interested in this trial, it could be a promising opportunity to explore a new treatment for managing PKU.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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 Criteria:
• • 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