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

Join a study for gene therapy in adults with Classic PKU.

Recruiting
18-55 years
All
Phase 1/2
This study is testing a new gene therapy called **NGGT002** for adults with **classic PKU**. PKU is a condition where the body can't break down an amino acid called phenylalanine (Phe) due to a problem with the PAH gene. **NGGT002** uses a special virus (rAAV8) to deliver a working copy of the PAH gene. Participants will receive one dose of **NGGT002** through an IV and will be monitored for 5 years to see if it's safe and if it works. In the first part of the study, three people will get a low dose, and then the dose might be increased or more people might get the low dose. In the second part, the same process will be repeated with a higher dose. **Key Points**: - **Study Duration**: 5 years with regular check-ups. - **Eligibility**: Adults aged 18-55 with classic PKU who are not responding to current treatments. - **Compensation**: Not mentioned. Participants must be willing to follow study procedures and use effective birth control if required.
Study details
    Phenylketonurias

NCT06332807

NGGT INC.

21 December 2024

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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