drugset / Trial / NCT07267065

AAV2-hAADC for Parkinson's Disease (PDCS-01)

NCT07267065 ↗

Phase 1 Not yet recruiting 9 enrolled Krzysztof Bankiewicz
Non-randomizedSequentialSingle-blindTreatment

Summary

This study is looking at whether a gene therapy called AAV2-hAADC is safe and may help people with Parkinson's Disease. AAV2-hAADC is intended to increase the levels of dopamine in your brain. It contains a virus called adeno-associated virus 2 (AAV2) that has been modified to carry the genetic code for an enzyme called Aromatic L-Amino Acid Decarboxylase, or AADC for short. In this study, AAV2-hAADC is delivered to two parts of the brain called the putamen and the caudate. Increasing the amount of AADC gene in these parts of the brain converts more levodopa into dopamine, the chemical that is lacking in PD.

Timeline

Start
2026-02-01
Primary completion
2028-08-01
Completion
2028-12-01

Drugs

EvaluationDrugModalityDoseRoute
Subject Eladocagene Exuparvovec Gene therapy (AAV / viral vector) 4.2e+12 vg Other
Subject Eladocagene Exuparvovec Gene therapy (AAV / viral vector) 1.3e+13 vg Other