REGENERATE-PD

International Phase II Study of Intracerebral AAV2-GDNF Gene Therapy in Adults with Moderate Parkinson’s Disease

22/05/2025 to 01/01/2029

Parkinson’s disease is a chronic, progressive neurodegenerative disorder characterized by the gradual loss of dopaminergic neurons in the brain. While established therapies such as pharmacological treatment, deep brain stimulation, and medication infusion pumps can effectively alleviate symptoms, they do not modify the underlying disease process. This international, randomized Phase II study investigates a novel gene therapy approach. Using an adeno-associated viral vector (AAV2), the gene encoding the neurotrophic factor GDNF (glial cell line-derived neurotrophic factor) is delivered to specific regions of the brain. The aim of this therapy is to support dopaminergic neurons and potentially influence the progression of Parkinson’s disease. The gene therapy is administered during a neurosurgical procedure via precise intracerebral delivery under intraoperative MRI guidance using the ClearPoint® system. The study evaluates the safety, tolerability, and potential clinical effects of the treatment. After screening, eligible participants are randomized in a 2:1 ratio and followed for a minimum of 18 months. The study is conducted at specialized centers in Germany, the United States, and Poland. The University Hospital Tübingen is one of only two participating study centers in Germany.