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GDNF Gene Therapy for Multiple System Atrophy
Sponsor: Brain Neurotherapy Bio, Inc.
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 with either a possible or probable diagnosis of Multiple System Atrophy.
Official title: Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
Key Details
Gender
All
Age Range
35 Years - 75 Years
Study Type
INTERVENTIONAL
Enrollment
9
Start Date
2023-10-03
Completion Date
2028-08
Last Updated
2026-01-30
Healthy Volunteers
No
Conditions
Interventions
AAV2-GDNF gene therapy
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Sham (Placebo) Surgery
Bilateral partial burr/twist holes without dural penetration
Locations (7)
University of California Irvine
Irvine, California, United States
Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Quest Research Institute
Farmington Hills, Michigan, United States
NYU Langone Health
New York, New York, United States
The Ohio State University Medical Center
Columbus, Ohio, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States