GDNF Gene Therapy for Multiple System Atrophy

Running, not enrolling · Phase 1 · Has a placebo group

Conditions studied: Multiple System Atrophy

In brief

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.

Key facts

Study ID

NCT04680065

Run by

Brain Neurotherapy Bio, Inc.

People needed

9

Starts

2023-10-03

Expected to finish

2028-08-01

Last updated by the study team

2026-01-30

Who can join

Age: 35 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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