NCT06432140

A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency

An Open, Dose-escalating and Dose Confirmation Trial to Evaluate the Safety and Efficacy of VGN-R09b by Intra Putamen Injection in Patients With Severe Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. · Industry
• Sex: All
• Age: 18 Months – 8 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

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 putamen. This is an open, dose-escalating and dose confirming study. The sponsor plans to explore two dose levels (6.0×1011vg and 1.28×1012vg) in dose-escalating phase (three subjects each cohort), then plans to have 10 subjects enrolled for dose confirmation phase. This study is to give evidence for the safety and efficacy of VGN-R09b treatment for patients with severe Aromatic L-amino acid decarboxylase (AADC) deficiency.

Conditions

• Aromatic L-amino Acid Decarboxylase Deficiency

Interventions

Type	Name	Description
GENETIC	VGN-R09b injection	Two levels of VGN-R09b will be injected into bilateral putamen in dose-escalating phase, and one dose level will be injected in dose confirming phase

Timeline

• Start date: 2024-07-02
• Primary completion: 2026-07-21
• Completion: 2030-09-20
• First posted: 2024-05-29
• Last updated: 2026-04-03

Locations

1 site across 1 country: China