Status: Recruiting
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
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.
Participants needed: 12
Trial details
Phase: Phase 1, Phase 2Age: 18-55Biological sex: AllType: InterventionalSponsor: NGGT INC.Updated: Dec 2, 2025Locations: 5
Eligibility criteria
Is willing and able to provide written, signed informed consent after the nature... [+14]
Subjects with PKU that is not due to PAH mutation [+25]