A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency

NCTID	NCT06432140 (View at clinicaltrials.gov)
Description	This trial includes dose-escalating part (phase 1) and dose confirming part, to prove the safety and efficacy of VGN-R09b to treat patients with severe AADC deficiency (Show More)
Development Status	Active
Indication	Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Disease Ontology Term	DOID:0090123
Compound Name	VGN-R09b
Compound Description	AADC + NTF
Sponsor	Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type	Industry
Recruitment Status	Not yet recruiting
Enrollment Count	16
Results Posted	Not Available

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	AAV9
Editor Type	none
Dose 1	6.0E11 vg
Dose 2	1.28E12 vg
Dose 3
Dose 4
Dose 5

No.of Trial Sites
Locations