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Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson’s Disease


Brief Summary:

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:

1. EITHER: Less than 5 years since clinical diagnosis of PD and mild to moderate

2. 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

– Inability to tolerate laying flat in an MRI or allergy to gadolinium


  • University of California Irvine, Irvine, California, United States, 92697
  • University of California San Francisco, San Francisco, California, United States, 94103
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