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Study identifier: NCT06332807 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

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

Condition: Phenylketonurias  ·  Sponsor: NGGT INC.

PhasePhase 1/Phase 2
Planned participants12
Who can joinAll sexes, 18 Years to 55 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Study Contact  ·  916-337-9683  ·  phedom@nggtbio.com

Jinpeng Zhu  ·  8651283912888  ·  jpzhu@nggtbio.com.cn

Always discuss trial participation with your own doctor first.

Locations (5)

Children's Hospital of Orange County HospitalOrange, California, United StatesRecruiting
University of MinnesotaMinneapolis, Minnesota, United StatesRecruiting
Atlantic Health SystemMorristown, New Jersey, United StatesRecruiting
University of Pittsburgh Medical CenterPittsburgh, Pennsylvania, United StatesRecruiting
University or Texas, Southwestern medical CenterDallas, Texas, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT06332807