Study LocatoR
Search Criteria: Men's Health; Studies that include males;
Studies Found: 1
pheNIX clinical study
The study will include a screening to make sure participants are healthy and are able to enroll in the study and receive a single administration of HMI-102, an investigational gene therapy for the treatment of adults with PKU. Following a single administration of HMI-102, participants will be observed periodically for 52 weeks to see if the gene therapy is helping the participant make their own PAH enzyme. Finally, participants will be seen less frequently for another four years to make sure that the new gene therapy is restoring active PAH enzyme activity to cells.