A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients

NCTID	NCT02852213 (View at clinicaltrials.gov)
Description	The overall objective of this study is to determine the safety and efficacy of AAV2-hAADC delivered to the substantia nigra pars compacta (SNc) and ventral tegmental area (VTA) in children with aromatic L-amino acid decarboxylase (AADC) deficiency. (Show More)
Indication	AADC Deficiency
Compound Name	AAV2-hAADC
Sponsor	Krzysztof Bankiewicz
Funder Type	Other
Status	Recruiting
Enrollment Count	42

Target Gene/Variant	DDC
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	CED
Drug Product Type	Viral vector
Target Tissue/Cell	Substantia nigra & ventral tegmental area
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	Dose: 1.3x10 11 vg (<160μL); concentration of 8.3x10^11 vg/mL
Dose 2	4.2 x 10^11 vg (160ul)
Dose 3	1.6 x 10^12 vg (60uL)
Dose 4	1.3 x 10^12 vg (500uL)
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
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