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
  • Phase

    Phase 1
  • Study Type

  • Status

  • Study Participants

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.
Study Started
Apr 01
Primary Completion
Dec 31
Study Completion
Jun 30
Last Update
Feb 24

Biological AAV2-GDNF

Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

Earlier stage PD Experimental

Later stage PD Experimental


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
No Results Posted