Phase 2 Trial of Brain-Delivered AAV2-GDNF Gene Therapy Versus Control Surgery in Adults Aged 45 to 75 With Moderate Parkinsons Disease

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a gene therapy called AAV2-GDNF delivered directly to the brain in adults with moderate Parkinsons Disease. This randomized, double-blinded, Phase 2 clinical trial compares the gene therapy to a control surgery in participants aged 45 to 75 who have had Parkinsons Disease for 4 to 10 years. The study aims to better understand the impact of this treatment on motor symptoms over time. Participants will receive either a single bilateral infusion of AAV2-GDNF into a brain region called the putamen or undergo a control surgery involving partial burr holes without dural penetration. These procedures are performed in a surgically controlled, blinded manner to compare outcomes between groups. This intervention phase is followed by ongoing assessments lasting up to 18 months. During the study, participants will be monitored regularly with assessments including the Parkinson Disease Motor Diary to track motor symptoms. Researchers will measure changes from baseline to 18 months to evaluate treatment effects. The study also includes safety monitoring and ensures participants maintain stable anti-parkinsonian medication throughout. Total participation may last over a year and a half, with detailed evaluations throughout the trial period.

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)