Phenylketonuria Clinical Trials
A listing of Phenylketonuria medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 185 clinical trials
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 …
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- 09 May, 2025
- 2 locations
AAV Gene Therapy Study for Subjects With PKU
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
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- 19 Feb, 2024
- 1 location
