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Clinical Trial Report

Gene Therapy Trial Report

Summary

An Early Clinical Trial to Evaluate VGN-R09b for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency.

NCTID NCT05765981 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Disease Ontology Term DOID:0090123
Compound Name VGN-R09b (AADC + NTF)
Sponsor Shanghai Jiao Tong University School of Medicine
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 6
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 Striatum
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed single dose
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Early phase1
Submit Date 2023-01-18
Completion Date 2029-02-20
Last Update 2023-03-13

Participation Criteria

Eligible Age 24 Months - 7 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations China

Regulatory Information

Has US IND False
FDA Designations
Recent Updates NMPA IND accepted 1/24/24; FDA IND accepted 7/26/24

Resources/Links

News and Press Releases