A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
Active, not recruiting · Phase 2 · NCT04903288
Conditions: AADC Deficiency
At a glance
- Research payments reported, 2025
- $445,233
- U.S. investigators named
- 3
- Sites paid
- 3
- Planned or actual enrollment
- 13
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- Eladocagene Exuparvovec (genetic)
- Dates
- Started 2021-05-12; completion 2028-04-30
- Results posted
- Yes
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-te…
From ClinicalTrials.gov, last updated 2026-04-24. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $445,233 in research payments for this study in 2025 (12 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Phillip Pearl, MD Child Neurology · Boston, MA
- Sudhakar Vadivelu, D.O Neurosurgery · Cincinnati, OH
- Daniel J. Curry, MD Neurosurgery · Houston, TX
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Cincinnati Children's Hospital Medical Center, OH $256,659
- Boston Children's Hospital, MA $105,277
- Baylor College of Medicine $82,097
- $1,200
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.