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

Sponsor:

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.

Criteria

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
UPDRS III OFF score

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
risk

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

Locations

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