Safety and Efficacy Study of NGGT002 in PKU Adult Subjects

NCT06061614 | Recruiting Early Phase 1

• 1 other identifier: NGGT002-P-2202
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Phenylketonurias

Keywords

Phenylalanine hydroxylase (PAH) Deficiency

Outcome Measures

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)

  Week 52

Secondary Outcomes (3)

• Plasma Phe levels

  Baseline Week 12, Week 28, Week 52

• Nature protein intake

  Baseline, Week 12, Week 28, and Week 52

• Food Phe intake

  Baseline, Week 12, Week 28, and Week 52

Study Arms (5)

Dose level 1

EXPERIMENTAL

Dose level 1 will be administered

Genetic: NGGT002 Injection

Dose level 2

EXPERIMENTAL

Dose level 2 will be administered

Genetic: NGGT002 Injection

Dose level 3

EXPERIMENTAL

Dose level 3 will be administered

Genetic: NGGT002 Injection

Dose level 4

EXPERIMENTAL

Dose level 4 will be administered

Genetic: NGGT002 Injection

Dose level 5

EXPERIMENTAL

Dose level 5 will be administered

Genetic: NGGT002 Injection

Interventions

NGGT002 Injection GENETIC

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

Dose level 1; Dose level 2; Dose level 3; Dose level 4; Dose level 5

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• Subjects are able to maintain their baseline diet throughout the study (regardless of dietary phenylalanine restriction), and willingness to follow the instruction of investigators to manage the diet for the duration of the trial;
• Subjects are required to obtain approval from the investigator prior to the use of any concomitant medications during the study period;
• Willingness and capable per Investigator opinion to comply with study procedures and requirements;
• Female participants of childbearing potential must have abstained from unprotected sexual intercourse for at least 14 days prior to dosing, and must have a documented negative serum hCG test between Day -7 and Day 0. All participants must be willing to use a highly effective method of contraception for at least 12 months following NGGT002.

You may not qualify if:

• 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
• Hematology values outside of the normal range (Hemoglobin \< 110 g/L (male), \< 100 g/L (female), white blood cells \< 3.0 × 10\^9/L, neutrophils \< 1.5 × 10\^9/L, platelet counts \< 100 × 10\^9/L;
• Hemoglobin A1c \> 6%, or fasting glucose \> 6.1 mmol/L;
• Clinically significant abnormalities in vital signs, physical examination findings, laboratory tests, or other assessments that, in the Investigator's judgment, are deemed unsuitable for study enrollment;
• Any contraindications to corticosteroid use or conditions potentially worsened by corticosteroids, as assessed by the Investigator, including but not limited to hypersensitivity to glucocorticoids, epilepsy, recent or unresolved bone fractures, ongoing wound healing, uncontrolled infections, or clinically significant osteoporosis;
• Subjects with a history of allergy to human serum albumin;
• All types of past and current malignancy;
• Severe diseases in the cardiovascular, respiratory, digestive tract, endocrine, kidney, blood, nervous, mental and other systems before screening;
• Subjects with history of live diseases, such as hepatitis, liver cirrhosis, liver cancer or other serious liver diseases;
• Subjects who participated in other clinical trails and took drugs within 3 months before screening;
• Other conditions that the Investigators deemed inappropriate for enrollment.

Sponsors & Collaborators

• The First Affiliated Hospital of Bengbu Medical University: lead

Study Sites (1)

The First Affiliated Hospital of Bengbu Medical College

Bengbu, Anhui, 233000, China

RECRUITING

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Huan Zhou, Doctor

CONTACT

008613665527160; zhouhuanbest@vip.163.com

Xiaoli Li, Master

CONTACT

008615215520890; 158169847@qq.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Single group

Study Record Dates

• First Submitted: September 25, 2023
• First Posted: September 29, 2023
• Study Start: March 30, 2023
• Primary Completion (Estimated): December 30, 2028
• Study Completion (Estimated): December 30, 2028
• Last Updated: March 4, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)