Actively Recruiting
An Open-Label, Multiple-Center, Phase I/II Dose Escalation Study for the Safety and Efficacy of NGGT002 in Adults With Classic Phenylketonuria
Led by NGGT INC. · Updated on 2025-12-02
12
Participants Needed
5
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and efficacy of NGGT002, a gene therapy using an adeno-associated viral vector carrying a functional copy of the human PAH gene, in adults with classic Phenylketonuria (PKU). This Phase 1/2, open-label, multiple-center study focuses on adults aged 18 to 55 with severe PAH deficiency and no residual enzyme activity. The aim is to assess the impact of NGGT002 in this population over a long-term period. Participants will receive a single intravenous infusion of NGGT002 at either a low or high dose. The study begins with a dose escalation phase where the first three subjects receive the low dose, followed by a decision to escalate or expand the cohort. If safety and efficacy are favorable, the study proceeds to a second part with the high dose in 3 to 6 subjects. The dosing is based on preclinical safety data to ensure the highest safe dose is selected. After receiving the gene therapy, participants will be followed for five years during which researchers will monitor safety by tracking adverse events, vital signs, ECGs, laboratory tests, and physical examinations. They will also measure changes in plasma phenylalanine levels and assess quality of life and protein intake. Participants are expected to record their diet and comply with study procedures. This long-term follow-up helps evaluate both the safety and the potential effects of NGGT002 over time.
CONDITIONS
Brief Title
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Willing and able to provide written consent before any research procedures
- Diagnosed with classic PKU caused by PAH mutations with no residual enzyme activity
- Adults aged 18 to 55 years at consent
- Intolerant or unresponsive to available PKU medical therapies
- Off PKU medications like Kuvan or Palynziq for at least 28 days before consent
- At least one documented phenylalanine measurement of 600 bcmol/L or higher on usual diet in past 6 months
- Willing to record diet and follow dietitian instructions during trial
- Capable and willing to comply with study procedures
- Women of childbearing potential must have negative pregnancy test and agree to effective contraception for at least 1 year after treatment; men must provide negative semen samples for AAV8 as required
You will not qualify if you...
- PKU not caused by PAH mutation
- Presence of anti-AAV8 neutralizing antibodies
- Abnormal liver function or blood test results exceeding specified limits on two tests
- Abnormal vital signs or clinical findings making participation unsafe
- Contraindications or risks related to corticosteroid use
- Active infections including hepatitis, HIV, tuberculosis, and syphilis
- History of liver diseases such as cirrhosis or non-alcoholic steatohepatitis within 6 months
- Any past or current cancer
- Liver fibrosis or other serious liver diseases detected by imaging
- Severe diseases in major organ systems
- Allergy to human albumin
- Substance use disorders
- Previous gene therapy treatments
- Recent investigational treatments within 3 months
- Elevated serum alpha-fetoprotein
- Other serious comorbidities or conditions deemed inappropriate by investigators
- Currently on PKU medications
- Weight over 120 kg
- Daily natural protein intake over 2 g/kg body weight
- Breastfeeding women
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 NGGT002 gene therapy through intravenous infusion at either a low or high dose, depending on cohort assignment.
Multiple visits over 52 weeks for dosing and assessments
Duration - Up to 5 years
Participants are monitored for safety and efficacy outcomes including adverse events, laboratory values, and plasma Phe concentration for up to 5 years after treatment.
Regular visits for assessments during Years 1 to 5
Trial Site Locations
Total: 5 locations
1
Children's Hospital of Orange County Hospital
Orange, California, United States, 92868
Actively Recruiting
2
University of Minnesota
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
3
Atlantic Health System
Morristown, New Jersey, United States, 07960
Actively Recruiting
4
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
5
University or Texas, Southwestern medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
Research Team
S
Study Contact
J
Jinpeng Zhu
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
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