GDNF Gene Therapy for Multiple System Atrophy
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Study 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
Primary outcome measure(s)
- The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations — 3 years
Number of TEAE and SAE's reported post-treatment.
Trial sites (7)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California Irvine | Irvine | California | |
| Parkinson's Disease and Movement Disorders Center of Boca Raton | Boca Raton | Florida | |
| Massachusetts General Hospital | Boston | Massachusetts | |
| Quest Research Institute | Farmington Hills | Michigan | |
| NYU Langone Health | New York | New York | |
| The Ohio State University Medical Center | Columbus | Ohio | |
| Vanderbilt University Medical Center | Nashville | Tennessee |
More Brain Neurotherapy Bio, Inc. trials in the USA
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04680065 on ClinicalTrials.gov ↗ ← All trials in the USA