This early Phase trial is to prove the safety and efficacy of VGN-R09b to treat patients with AADC deficiency.
Aromatic L-amino acid decarboxylase (AADC) is an enzyme responsible for the final step in the synthesis of neurotransmitters dopamine and serotonin. AADC deficiency is a rare genetic disorder. VGN-R09b is a kind of Gene therapy with adeno-associated virus (AAV) serotype 9 (AAV9) driven human AADC (hAADC) being injected directly into striatum. This is a single-center, open, dose-climbing investigator-sponsored early-stage clinical study that included a dose-climbing phase and a dose-expanding phase. This study is to give preliminary evidence for the safety and efficacy of VGN-R09b treatment for patients with Aromatic L-amino acid decarboxylase (AADC) deficiency.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
VGN-R09b will be injected into bilateral putamen by stereotactic surgery.(Through the SmartFlow Ventricular Cannula or the parenchymal injection tube actually used in the research center)
Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, No. 1678, Dongfang Road, Pudong New Area, Shanghai, China
RECRUITINGNumber of Adverse Events (AEs), Serious Adverse Events (SAEs)
Number of Adverse Events (AEs), Serious Adverse Events (SAEs)
Time frame: Week 52
Achievement Ratio of the four motor development milestones (Head control, Sit independently, Stand/stepping with support and Walk with minimal assistant)
Achievement Ratio of the four motor development milestones (Head control, Sit independently, Stand/stepping with support and Walk with minimal assistant) according to PDMS-II score.
Time frame: Week 52
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