NCT06332807 · NGGT INC.
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
What this study is about
This is a Phase 1/2, where both patients and doctors know the treatment given, multiple-center, gradually increasing doses and group of participants expansion study to evaluate the safety and effectiveness of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene.
View original scientific description
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.
Interventions
GENETIC
NGGT002
adeno-associated viral vector with human phenylalanine hydroxylase gene
Primary outcome measures
Incidence and severity of Adverse Events (AEs)
Time frame: Baseline to Week 52 and during Year 1 to 5
Incidence and severity of AEs, including serious AEs (SAEs) as assessed by CTCAE v5.0 of a single administration of NGGT002.
Change from baseline in clinical laboratory values
Time frame: Baseline to Week 52 and during Year 1 to 5
Change in chemistry values including liver function tests, hematology and urinalysis.
Change from baseline in 12-lead electrocardiograms (ECGs), vital signsand physical examinations
Time frame: Baseline to Week 52 and during Year 1 to 5
Subjects change from baseline in 12-lead electrocardiograms (ECGs), vital signs and physical examinations.
Change from baseline in Plasma Phe Concentration
Time frame: Baseline to Week 52 and during Year 1 to 5
To evaluate the efficacy in change of plasma Phe concentration of IV infusion of NGGT002 in adults with classic PKU at Week 12, Week 28, Week 52 and during Year 1 to 5.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Is willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any research-related procedures; a legally authorized representative may provide written consent and assent may be requested. 2. Male and female subjects with diagnosis of classic PKU, a condition characterized by severe PAH deficiency with confirmed PAH mutations predicted with no residual enzyme activity. A list of PAH mutations for classic PKU based on in vitro PAH activity (Himmelreich et al., 2018) and the genotype-phenotype correlation (Garbade et al., 2019) can be found in BIOPKU genotypes database (http://www.biopku.org/pah). 3. Adults aged 18-55 at the time of informed consent 4. Subjects intolerant or unresponsive to available medical therapies, such as Kuvan, Playnzip, etc. 5. Subjects who have been on medications, such as Kuvan, Palynziq, etc but have come off for medical reasons or the patient's decision at least 28 days prio
Where
- Orange, California
- Minneapolis, Minnesota
- Morristown, New Jersey
- Pittsburgh, Pennsylvania
- Dallas, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Dec 2, 2025 · Source of record for eligibility and locations