GDNF Gene Therapy for Multiple System Atrophy
Public ClinicalTrials.gov record NCT04680065. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04680065
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1
- Enrollment
- 9 participants
Conditions and interventions
Conditions
Interventions
- AAV2-GDNF gene therapy Biological
- Sham (Placebo) Surgery Procedure
Biological · Procedure
Eligibility (public fields only)
- Age range
- 35 Years to 75 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 3, 2023 (Actual)
- Primary completion
- Aug 2026 (Estimated)
- Completion
- Aug 2028 (Estimated)
- Last update posted
- Jan 30, 2026
2023 – 2028
United States locations
- U.S. sites
- 7
- U.S. states
- 7
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California Irvine | Irvine | California | 92697 | — |
| Parkinson's Disease and Movement Disorders Center of Boca Raton | Boca Raton | Florida | 33486 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Quest Research Institute | Farmington Hills | Michigan | 48334 | — |
| NYU Langone Health | New York | New York | 10016 | — |
| The Ohio State University Medical Center | Columbus | Ohio | 43210 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04680065, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 30, 2026 · Synced Oct 19, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04680065 live on ClinicalTrials.gov.