Trials / Active Not Recruiting
Active Not RecruitingNCT04680065
GDNF Gene Therapy for Multiple System Atrophy
Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
- Status
- Active Not Recruiting
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 9 (estimated)
- Sponsor
- Brain Neurotherapy Bio, Inc. · Industry
- Sex
- All
- Age
- 35 Years – 75 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| 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 |
Timeline
- Start date
- 2023-10-03
- Primary completion
- 2026-08-01
- Completion
- 2028-08-01
- First posted
- 2020-12-22
- Last updated
- 2026-01-30
Locations
7 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04680065. Inclusion in this directory is not an endorsement.