RecruitingEarly Phase 1NCT05765981

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

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.

Sponsor

Shanghai Jiao Tong University School of Medicine

Enrollment

6 participants

Start Date

Jan 30, 2023

Study Type

INTERVENTIONAL

Conditions

Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Summary

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

Eligibility

Min Age: 24 MonthsMax Age: 7 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called VGN-R09b for people with aromatic l-amino acid decarboxylase (aadc) deficiency. The study is currently recruiting participants at 1 location. People eligible for this study include aged 24 Months to 7 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGVGN-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

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NCT05765981