Actively Recruiting
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
Led by NGGT INC. · Updated on 2025-12-02
12
Participants Needed
5
Research Sites
311 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 an 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
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 informed consent before any study procedures
- Male or female adults aged 18-55 with classic PKU diagnosed by severe PAH deficiency and confirmed PAH mutations
- Intolerant or unresponsive to current PKU treatments such as Kuvan or Palynziq
- Off PKU medications like Kuvan or Palynziq for at least 28 days before consent
- At least one documented blood phenylalanine measurement of 600 bcmol/L or higher on usual diet within past 6 months
- Willing to record diet and follow dietitian instructions during the trial
- Capable of following study procedures as judged by the investigator
- Women of childbearing potential must have a negative pregnancy test and agree to use effective contraception from dosing until at least 1 year after
- Male participants must provide three consecutive semen samples negative for AAV8 after dosing
- Acceptable contraception methods include sterilization, condoms, hormonal contraception, intrauterine devices, or true sexual abstinence
You will not qualify if you...
- PKU not caused by PAH mutation
- Presence of anti-AAV8 neutralizing antibodies
- Abnormal liver function or hematology tests exceeding specified limits on two consecutive tests
- Abnormal vital signs or physical exam results considered clinically significant
- Contraindications or risk from corticosteroid use
- Active infections including hepatitis A, B, C, HIV, tuberculosis, or syphilis
- History of liver disease such as steatosis, fibrosis, NASH, cirrhosis, or biliary disease within 6 months (except Gilbert's syndrome)
- Any past or current malignancy
- Imaging showing liver fibrosis, cirrhosis, or serious liver diseases
- Severe diseases affecting major organs or systems
- Allergy to human albumin
- Substance use disorder (e.g., alcohol, heroin, amphetamines)
- Previous gene therapy treatment
- Use of investigational drugs within 3 months before screening
- Elevated serum alpha-fetoprotein
- Other serious comorbidities that could worsen with NGGT002
- Currently using PKU treatments like Kuvan or Palynziq
- Weight over 120 kg
- Consuming more than 2 grams of natural protein per kg of body weight daily
- Breastfeeding at time of study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
J
Jinpeng Zhu
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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