Back to search results

Gene Therapy for Adult Classic PKU (PHEdom)

This study is testing a gene therapy called NGGT002 for adults with classic Phenylketonuria (PKU), a condition where the body can't properly break down a substance called phenylalanine. NGGT002 is designed to deliver a working copy of the gene that helps process phenylalanine. Researchers want to see if NGGT002 is safe and how well it works. They will be looking for any side effects and changes in laboratory tests, heart readings (ECGs), vital signs, and physical exams over five years. You may be able to join if you are an adult between 18 and 55 years old with a diagnosis of classic PKU. The study plans to enroll 12 participants.

Study design
This is a Phase 1/2, open-label study, meaning both you and the study team will know what treatment you are receiving. It will involve a small number of participants, with 12 planned, and will test different doses of NGGT002.
What's involved
Participants will receive a single intravenous infusion of NGGT002 and will be followed for safety and how well the treatment works for five years.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety and efficacy for five years after receiving the treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06332807

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

Recruiting
PHASE1Ages 18–55InterventionalTreatment
NGGT INC.
~12 participants
Updated 2026-08-18 on ClinicalTrials.gov
What's tested:NGGT002

At a glance

Recruiting sites
5 of 5 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence and severity of Adverse Events (AEs)
Measured over Baseline to Week 52 and during Year 1 to 5
+3 more outcomes measured

Conditions

Phenylketonurias

Explore related trials

PhenylketonuriaPAH Gene

Where it's being run

5 sites across 5 states
California1
Minnesota1
New Jersey1
Pennsylvania1
Texas1

Who to contact

Study Contact
[email protected]
916-337-9683
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

documented vasectomy or permanent sterilization
condom
combined (estrogen and progestogen-containing) hormonal contraception (oral, intravaginal or transdermal)
progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable)
intrauterine device
intrauterine hormone-releasing system
sexual abstinence is acceptable only as true abstinence and when in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, hypothermal, post-ovulation) is not acceptable as a form of abstinence.

Exclusion

Alanine aminotransferase (ALT) \>1.5×ULN and/or aspartate aminotransferase (AST) \>1.5×ULN
Alkaline phosphatase (ALP) \>1.5×ULN
Total bilirubin (TBil) \>1.5×ULN, direct bilirubin \>1.5×ULN
International normalized ratio (INR) \> 1.5
Blood creatinine (Scr) \>1.5×ULN
Hematology values outside of the normal range (Hemoglobin \<110 g/L (male), \<100 g/L (female), white blood cell \<3.0×10\^9/L, neutrophil \<1.5×10\^9/L, platelet \<100×10\^9/L)
Hemoglobin A1c \>6% or fasting glucose \>6.1 mmol/L 4. At the time of screening, abnormal vital signs (i.e. Temperature\<36.3°C or \>37.4°C; Blood pressure\<100/60 mmHg or \>130/80 mmHg; heart rate \<60/min or\>100/min; respiratory rate \<12/min or \>18/min; oxygen saturation\<95%), physical examination, laboratory tests, or other related results that have clinical significance, and the researchers believe they are unsuitable for enrollment. 5. Contraindications to corticosteroid use or possible deterioration of corticosteroid use assessed and determined by the Investigator. 6. Active infection with hepatitis A virus (HAV ribonucleic acid \[RNA\] positive), active or occult hepatitis B virus infection (positive HBV-DNA or anti-HBc positive with negative hBsAg, HBV surface antigen), active infection with hepatitis C virus (HCV RNA positive), infection with the human immunodeficiency virus (HIV) as measured by antibodies to HIV-1 and HIV-2, active or latent infection with tuberculosis (TB) measured by Quantiferon Gold, infection with syphilis by rapid plasma regainn (RPR) and/or serum syphilis antibody, treponema pallidum particle agglutination (TPPA). 7. Subjects with history of liver disease such as clinically significant steatosis, fibrosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, biliary disease within 6 months of informed consent; except for Gilbert's syndrome. 8. All types of past and current malignancy 9. Imaging (liver ultrasound) proved the existence of Liver fibrosis, liver cirrhosis and other serious liver diseases 10. Severe diseases in the cardiovascular, respiratory, digestive tract, endocrine, kidney, blood, nervous, mental and other systems before screening. 11. History of allergy to Albumin (Human) 12. The subjects who have Substance Use Disorder (for example alcohol, heroin, amphetamine, etc) 13. The subjects who have received any gene therapy in the past, regardless of when it was administered. 14. The subjects who have received any investigational treatment and took drugs within 3 months before screening (or 5 half-lives, if longer) 15. Subjects with elevated circulating serum alpha-fetoprotein (AFP) 16. Other conditions that the Investigators deemed inappropriate for enrollment, such as PKU severe comorbidities and conditions (i.e. renal insufficiency or kidney failure, osteoporosis, anemia, acid reflux or gastro-esophageal ulcer, major depression, epilepsy, etc.), which may be deteriorated with the potential risks of NGGT002. 17. Subjects who are presently on available medications for the treatment of PKU, such as Kuvan, Palynziq, etc. 18. Subjects who weight over 120 Kg 19. Subjects who consume too much natural protein (\>2 g/Kg body weight/day) in their daily diet 20. Breastfeeding subjects will not be included in the study

What this trial measures

  • Incidence and severity of Adverse Events (AEs)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 valuesBaseline 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 examinationsBaseline 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 ConcentrationBaseline 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.