Condition: Phenylketonurias · Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd.
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 a 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.
Huan Zhou, PhD · 8613665527160 · zhouhuanbest@vip.163.com
Always discuss trial participation with your own doctor first.
| First Affiliated Hospital of Bengbu Medical College | Bengbu, Anhui, China | Recruiting |
| Xinhua Hospital Affifiated to Shanghai Jiao Tong University School of Medicine | Shanghai, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06687733