NCT04903288

A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

An Open-Label Trial to Address the Safety of the SmartFlow MR-Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Subjects

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 1 Year – 17 Years
• Healthy volunteers: Not accepted

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.

Conditions

• AADC Deficiency

Interventions

Type	Name	Description
GENETIC	Eladocagene Exuparvovec	Four 0.08 milliliters (mL) infusions at a dose of 0.45×10\^11 vg and a volume of 80 microliters (μl) per site to 4 sites (2 per putamen), for the total dose of 1.8×10\^11 vg and a total volume of 320 μl per participant.

Timeline

• Start date: 2021-05-12
• Primary completion: 2023-05-22
• Completion: 2028-04-30
• First posted: 2021-05-26
• Last updated: 2026-02-25
• Results posted: 2025-01-01

Locations

6 sites across 3 countries: United States, Israel, Taiwan

Regulatory

• FDA-regulated drug study
• FDA-regulated device study