for people ages 35-75 (full criteria)
at San Francisco, California and other locations
study started
estimated completion



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.

Official Title

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


Parkinson's Disease PD Neurotrophic factor Growth factor Glial cell line-derived neurotrophic factor GDNF AAV Gene therapy Parkinson Disease AAV2-GDNF


For people ages 35-75

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


  • University of California San Francisco not yet accepting patients
    San Francisco California 94103 United States
  • The Ohio State University Medical Center accepting new patients
    Columbus Ohio 43210 United States


accepting new patients
Start Date
Completion Date
Brain Neurotherapy Bio, Inc.
Phase 1
Study Type
Last Updated