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

NCTID	NCT04903288 (View at clinicaltrials.gov)
Description	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. (Show More)
Indication	AADC Deficiency
Compound Name	UPSTAZA (eladocagene exuparvovec)
Sponsor	PTC Therapeutics
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	13

Target Gene/Variant	DDC
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intraparenchymal
Drug Product Type	Viral vector
Target Tissue/Cell	Putamen
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	1.8×10^11 vg/dose, 320 μl/dose
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	6
Locations	United States,Taiwan,Israel

Has US IND	True
Recent Updates	BLA Submitted: Priority Review Action Date (11/13/24)