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RECRUITING
NCT05765981
EARLY_PHASE1

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

Sponsor: Shanghai Jiao Tong University School of Medicine

View on ClinicalTrials.gov

Summary

This early Phase trial is to prove the safety and efficacy of VGN-R09b to treat patients with AADC deficiency.

Official title: An Open, Dose-escalating Early Clinical Trial to Evaluate the Tolerability, Safety, and Efficacy of VGN-R09b by Intra Striatum Injection in Patients With Aromatic L-amino Acid Decarboxylase (AADC) Deficiency.

Key Details

Gender

All

Age Range

24 Months - 7 Years

Study Type

INTERVENTIONAL

Enrollment

6

Start Date

2023-01-30

Completion Date

2029-02-20

Last Updated

2023-03-13

Healthy Volunteers

No

Interventions

DRUG

VGN-R09b

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)

Locations (1)

Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, No. 1678, Dongfang Road, Pudong New Area, Shanghai, China