GDNF Gene Therapy for Multiple System Atrophy

Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy

The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.

Inclusion Criteria:

• Male and female 35-75 years of age (inclusive)
• Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and > 30 years of age
• Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years
• Stable anti-parkinsonian medication regimen
• Ability to walk a distance of 25 feet with or without an assistive device

Exclusion Criteria:

• Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases
• Presence of dementia, psychosis, substance abuse or poorly controlled depression
• Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities
• History of cancer or poorly controlled medical conditions that would increase surgical risk
• Received investigational agent within 12 weeks
• Inability to tolerate laying flat in an MRI and/or allergy to gadolinium

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.