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ACTIVE NOT RECRUITING
NCT04680065
PHASE1

GDNF Gene Therapy for Multiple System Atrophy

Sponsor: Brain Neurotherapy Bio, Inc.

View on ClinicalTrials.gov

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

Interventions

BIOLOGICAL

AAV2-GDNF gene therapy

Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

PROCEDURE

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