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Eladocagene Exuparvovec

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

Ensayo de PTC THERAPEUTICS, INC. en AADC Deficiency (del registro, en inglés).

Fase

Fase 2

Estado

En marcha, sin reclutar

Participantes

13

previstos

Centros

6

Fin del objetivo primario

may 2023

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en may 2021.

Qué mide

Change From Baseline in HVA Metabolite Level at the End of the Trial Phase (Baseline (Day 1), Week 8) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis 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.

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Ficha completa en ClinicalTrials.gov (NCT04903288), actualizada en abr 2026.