Actively Recruiting
A Clinical Study for the Safety and Efficacy of IV Infusion of NGGT002 in the Treatment of Phenylketonuria
Led by The First Affiliated Hospital of Bengbu Medical University · Updated on 2026-03-04
15
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and effectiveness of NGGT002 gene therapy in adults diagnosed with Phenylketonuria (PKU) caused by mutations in the PAH gene. This early phase 1, single-center, open-label, non-randomized study aims to understand how a single intravenous dose of NGGT002 affects PKU by gradually increasing doses to determine the best treatment level. NGGT002 is given through an intravenous infusion starting at Dose Level 1, followed by stepwise dose escalation up to Dose Level 5 as safety and efficacy data are reviewed. After each dose cohort, researchers decide whether to expand that group or move to the next dose level. All subjects receive a single administration of NGGT002 and are monitored closely. Participants will be followed for safety and efficacy over five years after receiving NGGT002. Researchers will assess adverse events during the first year and measure plasma phenylalanine levels, natural protein intake, and dietary phenylalanine intake at various points up to week 52. The study monitors participants’ diet and health status throughout, with long-term follow-up to understand lasting effects of the gene therapy.
CONDITIONS
Brief Title
Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Voluntarily sign informed consent form
- Male or female adult aged 18 years or older
- Diagnosed with PKU caused by confirmed PAH gene mutation according to Clinical Practice Guidelines for Phenylketonuria, 2020 Edition
- Blood phenylalanine (Phe) concentration of at least 600 bcmol/L once within 2 years prior to screening, with one measurement confirmed within six months before enrollment
- Able to maintain baseline diet throughout the study and willing to follow investigators' diet instructions
- Obtain investigator approval before any concomitant medication use during study
- Willing and able to comply with study procedures and requirements
- Female participants of childbearing potential must have abstained from unprotected sex for at least 14 days prior to dosing and have a documented negative serum hCG test between Day -7 and Day 0
- All participants must agree to use a highly effective contraception method for at least 12 months after NGGT002 administration
You will not qualify if you...
- Anti-AAV8 neutralizing antibody titer greater than 1:10
- Prior gene therapy treatment
- Positive for hepatitis B surface antigen, hepatitis C antibody, HIV antibody, or treponema pallidum-specific antibody
- Abnormal liver function tests exceeding 1.5 times upper limit of normal for ALT, AST, ALP, or total bilirubin, or INR over 1.3
- Hematology values outside normal range (Hemoglobin <110 g/L for males, <100 g/L for females; white blood cells <3.0 d7 10^9/L; neutrophils <1.5 d7 10^9/L; platelet count <100 d7 10^9/L)
- Hemoglobin A1c over 6% or fasting glucose over 6.1 mmol/L
- Significant abnormalities in vital signs, physical exams, or lab tests deemed unsuitable by Investigator
- Contraindications to corticosteroid use or conditions worsened by corticosteroids, including hypersensitivity, epilepsy, recent fractures, wound healing issues, infections, or osteoporosis
- History of allergy to human serum albumin
- Past or current malignancy of any type
- Severe diseases affecting cardiovascular, respiratory, digestive, endocrine, kidney, blood, nervous, mental, or other systems
- History of serious liver diseases such as hepatitis, cirrhosis, or liver cancer
- Participation in other clinical trials with drug use within 3 months prior to screening
- Other conditions Investigator judges inappropriate for enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive a single intravenous infusion of NGGT002 gene therapy with dose escalation through five levels to evaluate safety and efficacy.
Baseline visit and follow-up visits at Week 12, Week 28, and Week 52
Trial Site Locations
Total: 1 location
1
The First Affiliated Hospital of Bengbu Medical College
Bengbu, Anhui, China, 233000
Actively Recruiting
Research Team
H
Huan Zhou, Doctor
X
Xiaoli Li, Master
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
5
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