Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
Who is this study for? Patients with multiple system atrophy
What treatments are being studied? AAV2-GDNF gene therapy
Status: Recruiting
Location: See all (7) locations...
Intervention Type: Biological, Procedure
Study Type: Interventional
Study Phase: Phase 1
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 35
Maximum Age: 75
Healthy Volunteers: f
View:
• 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
Locations
United States
California
University of California Irvine
RECRUITING
Irvine
Florida
Parkinson's Disease and Movement Disorders Center of Boca Raton
RECRUITING
Boca Raton
Massachusetts
Massachusetts General Hospital
ACTIVE_NOT_RECRUITING
Boston
Michigan
Quest Research Institute
RECRUITING
Farmington Hills
New York
NYU Langone Health
RECRUITING
New York
Ohio
The Ohio State University Medical Center
ACTIVE_NOT_RECRUITING
Columbus
Tennessee
Vanderbilt University Medical Center
RECRUITING
Nashville
Contact Information
Primary
Akash Pandhare, MD
askfirst@askbio.com
336-265-2790
Backup
Christian Urrea, MD
askfirst@askbio.com
336-265-2790
Time Frame
Start Date: 2023-10-03
Estimated Completion Date: 2028-12
Participants
Target number of participants: 9
Treatments
Experimental: Active Treatment
Sham_comparator: Placebo Surgery
Authors
Related Therapeutic Areas
Sponsors
Leads: Brain Neurotherapy Bio, Inc.