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NCT04903288 · ClinicalTrials.gov registry record · Phase 2
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
A Phase 2 study of AADC Deficiency, sponsored by PTC Therapeutics.
- Active
- Registry status
- Phase 2
- Development phase
- 13
- Enrollment target
- 6
- Study locations
NCT04903288: Active Phase 2 study of AADC Deficiency, sponsored by PTC Therapeutics.
NCT04903288 is a Phase 2 study of AADC Deficiency that is active but no longer recruiting, run by PTC Therapeutics. The registered enrollment target is 13 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (90% lower). The trial reports 6 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04903288, a Phase 2 study of AADC Deficiency, is active but no longer recruiting, sponsored by PTC Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 13 participants
- Enrollment target
- 6
- Study locations
Study Summary
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.
Primary Outcome
HVA is a main metabolite of dopamine and HVA CSF levels are recognized as a proxy for dopamine levels in the brain.
Conditions Studied
Interventions
- GENETIC Eladocagene Exuparvovec
Study Locations (6)
Other
- Chaim Sheba Medical Center - Ramat Gan
- National Taiwan University Hospital, Department of Pediatrics and Medical Genetics - Taipei
Massachusetts
- Boston Children's Hospital - Boston
North Carolina
- Duke University Hospital - Durham
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Texas
- Texas Children's Hospital - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2021-05-12 |
| Est. Completion | 2028-04-30 |
| Phase | Phase 2 |
What the registry record for NCT04903288 still lists
NCT04903288 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 13 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (90% lower).
The record links to 1 condition, with AADC Deficiency appearing as the primary indexed condition, and to 1 intervention - of which Eladocagene Exuparvovec is the first listed.
NCT04903288 lists 6 locations in 5 states (Other, Massachusetts, North Carolina).
Frequently Asked Questions
What is clinical trial NCT04903288 about?
NCT04903288 is a clinical study titled "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® magn...
What is the current status of trial NCT04903288?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 13 participants. The study started on 2021-05-12. Estimated completion is 2028-04-30.
What conditions does trial NCT04903288 study?
This clinical trial studies the following conditions: AADC Deficiency.
What interventions are being tested in trial NCT04903288?
The interventions under investigation include: Eladocagene Exuparvovec (GENETIC).
Who is sponsoring clinical trial NCT04903288?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04903288 being conducted?
This trial has 6 study locations across Massachusetts, North Carolina, Ohio, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for AADC Deficiency
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04903288's enrollment target sits among peer trials
13 1947th of 2000 higher than 53 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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