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Summary
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 (ESTIMATED)
Results Posted
Not Available
Therapy Information
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
Study Record Dates
Current Stage
Phase1
Submit Date
2024-05-17
Completion Date
2030-09
Last Update
2024-06-03
Participation Criteria
Eligible Age
18 Months - 8 Years
Standard Ages
Child
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
Locations
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
FDA IND accepted 7/26/24
Resources/Links
News and Press Releases
VGN-R09b, China's first domestically developed gene therapy drug for the treatment of aromatic L-amino acid decarboxylase deficiency, completed the first subject administration in a registered study
Related NCTID
Early Phase 1: NCT05765981