Actively Recruiting
Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
Led by NGGT (Suzhou) Biotechnology Co., Ltd. · Updated on 2024-11-14
18
Participants Needed
2
Research Sites
365 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is a rAAV8 based vector carrying a functional copy of the human PAH gene. Participants 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 cPKU Adult Subjects
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Voluntarily agree to participate and sign informed consent
- Any gender; must carry biallelic pathogenic or likely pathogenic variants in the PAH gene
- Adults aged 18 to 55 years
- At least two blood phenylalanine concentrations ≥600 µmol/L in past 24 months, with one measurement within 6 months before screening
- Willing and able to manage their diet
- Willing and able to comply with study procedures per investigator
- Women of childbearing potential must have negative serum HCG within 7 days before dosing and agree to use effective contraception for at least one year after treatment
You will not qualify if you...
- Presence of anti-AAV8 neutralizing antibodies (≥1:5)
- Disease well-controlled with existing therapies like Sapropterin or Pegvaliase
- Abnormal lab tests exceeding specified limits before dosing, including elevated liver enzymes, bilirubin, creatinine, abnormal blood counts, or glucose/HbA1c levels
- Clinically significant abnormal vital signs or examination findings making subject unsuitable
- Contraindications to corticosteroids or conditions that could worsen with their use
- Active or past infections: hepatitis A, B, C, HIV, syphilis, tuberculosis
- Significant liver disease history within 6 months except Gilbert's syndrome
- History of malignant tumors
- Severe liver disease evidence on imaging
- History of serious systemic diseases before screening
- Allergy to human serum albumin
- History of substance abuse
- Previous gene therapy treatment
- Participation in other investigational drug trials within 3 months prior to screening
- Elevated Alpha-fetoprotein (AFP)
- Severe comorbidities related to PKU such as renal failure, osteoporosis, anemia, major depression, epilepsy
- Weight over 100 kg
- Excessive natural protein intake (>2 g/kg/day)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
First Affiliated Hospital of Bengbu Medical College
Bengbu, Anhui, China
Actively Recruiting
2
Xinhua Hospital Affifiated to Shanghai Jiao Tong University School of Medicine
Shanghai, China
Actively Recruiting
Research Team
H
Huan Zhou, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here