AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) (NCT06332807)
An Open-Label, Multiple-Center, Phase I/II Dose Escalation Study for the Safety and Efficacy of NGGT002 in Adults With Classic Phenylketonuria
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (16 items) and avoid the listed exclusions (28 items).
Location: Children's Hospital of Orange County Hospital — Orange, California
Age range: 18 Years to 55 Years
🧪 Interventions in This Study
- NGGT002 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- Subjects with PKU that is not due to PAH mutation
- Presence of anti-AAV8 neutralizing antibodies
- Prior to dosing, subjects exceed the limit of any of the following liver function and hematology tests in two consecutive blood laboratory tests:
- 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
- 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.
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (5 Sites)
- Children's Hospital of Orange County Hospital — Orange, California
- University of Minnesota — Minneapolis, Minnesota
- Atlantic Health System — Morristown, New Jersey
- University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania
- University or Texas, Southwestern medical Center — Dallas, Texas
📞 Contact the Study Coordinator
Phone: (916) 337-9683
Email: phedom@nggtbio.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
- 💵 Compensation indicated: Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.