Status:
ACTIVE_NOT_RECRUITING
GDNF Gene Therapy for Multiple System Atrophy
Lead Sponsor:
Brain Neurotherapy Bio, Inc.
Conditions:
Multiple System Atrophy
Eligibility:
All Genders
35-75 years
Phase:
PHASE1
Brief Summary
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 wit...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
October 3 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2028
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT04680065
Start Date
October 3 2023
End Date
August 1 2028
Last Update
July 17 2025
Active Locations (7)
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1
University of California Irvine
Irvine, California, United States, 92697
2
Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, United States, 33486
3
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
4
Quest Research Institute
Farmington Hills, Michigan, United States, 48334