Clinical trials: AADC Deficiency
AADC Deficiency
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
This study will have a trial phase, extension phase, and a long-term extension phase. The primary objectives of the trial phase are to assess the pharmacodynamics (PD) of eladocagene exuparvovec treatment by evaluation of…
- Trial name
- NCT04903288
- Testing
- Eladocagene Exuparvovec
- Where
- 6 sites in United States, Israel and 1 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics