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.
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Who can participate?
Age: Over 18 years old
Volunteers: Volunteers with special conditions
Location: In Person
- Are diagnosed with PKU and 18-55 years old
- Have classic PKU due to phenylalanine hydroxylase (PAH) deficiency where the PAH gene is not working properly
- Are willing and able to maintain your existing diet
- Are able to take oral steroids for a period of time
- Are willing to commit to all required study visits and tests for the entire duration of the trial
Will I be paid for my time?