GDNF Gene Therapy for Parkinson's Disease
Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease
The objective of this Phase 1b investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a recent or a long-standing diagnosis of PD.
Key Inclusion Criteria: Male and female adults 35-75 years of age (inclusive) Diagnosed with Parkinson's disease Modified Hoehn and Yahr stage I-III OFF medication Time since receiving a clinical diagnosis of PD and disease severity consistent with one of the following: EITHER: Less than 5 years since clinical diagnosis of PD and mild to moderate UPDRS III OFF score OR: At least 4 years since clinical diagnosis of PD and moderate to severe UPDRS III OFF score Responsiveness to levodopa Key Exclusion Criteria: Atypical parkinsonism Severe dyskinesia Presence of dementia, psychosis, substance abuse or qualify as "severe depression" Prior brain surgery (i.e. deep brain stimulator or DBS implantation) or other brain imaging abnormalities Receiving an investigational drug History of cancer or poorly controlled medical conditions that would increase surgical risk Inability to tolerate laying flat in an MRI or allergy to gadolinium