Actively Recruiting
Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
Led by The First Affiliated Hospital of Bengbu Medical University · Updated on 2026-03-04
15
Participants Needed
1
Research Sites
300 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official 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 and female adults aged 18 years or older with PKU diagnosed by confirmed PAH gene mutation
- Blood phenylalanine concentration of 600 µmol/L or higher at least once within 2 years before screening, with one measurement confirmed within 6 months prior to enrollment
- Ability to maintain baseline diet throughout the study and willingness to follow diet management instructions
- Approval from investigator before using any medications during the study
- Willingness and capability to comply with study procedures
- Female participants of childbearing potential must have abstained from unprotected sex for at least 14 days before dosing and have a negative serum hCG test between Day -7 and Day 0
- All participants must agree to use a highly effective contraceptive method for at least 12 months after NGGT002 administration
You will not qualify if you...
- Presence of anti-AAV8 neutralizing antibody greater than 1:10
- Prior gene therapy treatment
- Positive for hepatitis B surface antigen, hepatitis C antibody, HIV antibody, or syphilis antibody
- Abnormal liver function tests: ALT or AST over 1.5 times upper limit, ALP over 1.5 times upper limit, total bilirubin over 1.5 times upper limit, or INR over 1.3
- Abnormal blood counts: hemoglobin below 110 g/L (male) or 100 g/L (female), white blood cells below 3.0 x10^9/L, neutrophils below 1.5 x10^9/L, or platelets below 100 x10^9/L
- Hemoglobin A1c above 6% or fasting glucose above 6.1 mmol/L
- Any significant abnormalities in vital signs, physical exam, labs, or assessments that make participation unsuitable
- Contraindications to corticosteroid use or conditions worsened by corticosteroids, including hypersensitivity, epilepsy, recent fractures, ongoing wounds, uncontrolled infections, or significant osteoporosis
- History of allergy to human serum albumin
- Any past or current cancer
- Severe diseases affecting cardiovascular, respiratory, digestive, endocrine, kidney, blood, nervous, or mental systems
- History of serious liver diseases such as hepatitis, cirrhosis, or liver cancer
- Participation in other drug clinical trials within 3 months before screening
- Any other conditions judged inappropriate by investigators for enrollment
AI-Screening
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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
CONTACT
X
Xiaoli Li, Master
CONTACT
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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