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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 homovanillic acid (HVA) levels and to assess the safety of the SmartFlow® magnetic resonance (MR) Compatible Ventricular Cannula for administering eladocagene exuparvovec to pediatric participants with aromatic L-amino acid decarboxylase (AADC) deficiency. The extension phase is designed to capture additional clinical information for eladocagene exuparvovec through study evaluations, changes in motor development, AADC-specific symptoms, and other PD measures. The long-term extension phase is designed to capture long-term safety and efficacy data from participants treated with eladocagene exuparvovec.
Details
| Lead sponsor | PTC Therapeutics |
|---|---|
| Phase | PHASE2 |
| Status | ACTIVE_NOT_RECRUITING |
| Enrolment | 13 |
| Start date | Wed May 12 2021 00:00:00 GMT+0000 (Coordinated Universal Time) |
| Completion | Sun Apr 30 2028 00:00:00 GMT+0000 (Coordinated Universal Time) |
Conditions
- AADC Deficiency
Interventions
- Eladocagene Exuparvovec
Countries
Taiwan, United States, Israel