GDNF Gene Therapy for Multiple System Atrophy
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
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
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.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study Details
Contition
Multiple System Atrophy
Age
35 - 75
Phase
PHASE1
Participants Needed
9
Est. Completion Date
Dec 31, 2028
Treatment Type
INTERVENTIONAL
Sponsor
Brain Neurotherapy Bio, Inc.
ClinicalTrials.gov NCT Identifier
NCT04680065
Study Number
MSA-101
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