Early Phase 1 Trial of VGN-R09b Gene Therapy by Brain Injection for Children With Aromatic L-amino Acid Decarboxylase Deficiency

What this Trial Is About

The trial focuses on patients with Aromatic L-amino acid decarboxylase AADC deficiency, a rare genetic disorder affecting the production of important neurotransmitters dopamine and serotonin. Researchers are evaluating the safety and effectiveness of VGN-R09b, a gene therapy delivered directly into the brain, to improve symptoms related to this condition. This early phase clinical study aims to provide preliminary evidence for this treatments impact on patients with AADC deficiency. Participants will receive VGN-R09b through injections into the bilateral putamen area of the brain using stereotactic surgery. The study includes a dose-escalation phase followed by a dose-expansion phase to assess different dose levels. This open-label, single-center trial monitors patients after receiving the gene therapy to understand its effects and tolerability. During the study, participants will be closely monitored for adverse events and their motor development milestones, such as head control, sitting independently, standing or stepping with support, and walking with minimal assistance, over a period of 52 weeks. Researchers will conduct various assessments and follow-ups to track the treatments safety and effectiveness, with the overall participation lasting about one year.

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