Safety and Efficacy Study of NGGT002 in PKU Adult Subjects

Phenylketonurias Clinical Trial

Official title:

A Clinical Study for the Safety and Efficacy of IV Infusion of NGGT002 in the Treatment of Phenylketonuria

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06061614
• Other study ID #: NGGT002-P-2202
• Status: Recruiting
• Phase: Early Phase 1
• Start date: March 30, 2023
• Est. completion date: December 30, 2028

Study information

• Verified date: February 2024
• Source: First Affiliated Hospital Bengbu Medical College
• Contact: Huan Zhou, Doctor
• Phone: 008613665527160
• Email: zhouhuanbest[@]vip.163.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 human PAH enzyme in adult Phenylketonuria (PKU) subjects with PAH deficiency. All participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.

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 mutations. NGGT002 will be administered through intravenous infusion. Dose escalation will be initiated from the low dose of NGGT002. After evaluating the safety and efficacy data, a decision will be made regarding the cohort expansion or the escalation to the next dose level. The same process will be followed for subsequent dose cohorts.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 9
• Est. completion date: December 30, 2028
• Est. primary completion date: December 30, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

Genetic:
• NGGT002
NGGT002

Locations

Country	Name	City	State
China	The First Affiliated Hospital of Bengbu Medical College	Bengbu	Anhui

Sponsors (1)

Lead Sponsor	Collaborator
First Affiliated Hospital Bengbu Medical College

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	total protein intake	Change from baseline in total protein intake	W12, W28, W52
Primary	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)	Week 52
Secondary	plasma Phe levels	Change from baseline in mean plasma Phe levels	W12, W28, W52