A Phase 1 gene therapy study of scAAV9/AGA in Aspartylglucosaminuria (AGU)
Not Yet Recruiting Phase 1,Phase 2 NCT07530796
Study Description
A first-in-human study testing a new gene therapy treatment for children and adults with Aspartylglucosaminuria (AGU)
Study Details
- Registry ID:
- NCT07530796
- Conditions:
- Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency
- Phase:
- Phase 1,Phase 2
- Target Enrollment:
- 9 participants
- Start Date:
- Age Range:
- 4–45 years
- Healthy Volunteers:
- No
Interested in Participating?
Check your eligibility and learn more about this study on TrialScreen.