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NCT04680065 · ClinicalTrials.gov registry record · Phase 1
GDNF Gene Therapy for Multiple System Atrophy
A Phase 1 study of Multiple System Atrophy, sponsored by Brain Neurotherapy Bio.
- Active
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
- 7
- Study locations
NCT04680065: Active Phase 1 study of Multiple System Atrophy, sponsored by Brain Neurotherapy Bio.
NCT04680065 is a Phase 1 study of Multiple System Atrophy that is active but no longer recruiting, run by Brain Neurotherapy Bio. The registered enrollment target is 9 participants, below the 268-participant average among 30 other Multiple System Atrophy trials with a reported enrollment target (97% lower). The trial reports 7 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04680065, a Phase 1 study of Multiple System Atrophy, is active but no longer recruiting, sponsored by Brain Neurotherapy Bio.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
- 7
- Study locations
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.
Primary Outcome
Number of TEAE and SAE's reported post-treatment.
Conditions Studied
Interventions
- BIOLOGICAL AAV2-GDNF gene therapy
- PROCEDURE Sham (Placebo) Surgery
Study Locations (7)
California
- University of California Irvine - Irvine
Florida
- Parkinson's Disease and Movement Disorders Center of Boca Raton - Boca Raton
Massachusetts
- Massachusetts General Hospital - Boston
Michigan
- Quest Research Institute - Farmington Hills
New York
- NYU Langone Health - New York
Ohio
- The Ohio State University Medical Center - Columbus
Tennessee
- Vanderbilt University Medical Center - Nashville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2023-10-03 |
| Est. Completion | 2028-08 |
| Phase | Phase 1 |
What the registry record for NCT04680065 still lists
NCT04680065 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 268-participant average among 30 other Multiple System Atrophy trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Multiple System Atrophy appearing as the primary indexed condition, and to 2 interventions - of which AAV2-GDNF gene therapy is the first listed.
NCT04680065 lists 7 locations in 7 states (California, Florida, Massachusetts).
Frequently Asked Questions
What is clinical trial NCT04680065 about?
NCT04680065 is a clinical study titled "GDNF Gene Therapy for Multiple System Atrophy". 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.
What is the current status of trial NCT04680065?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2023-10-03. Estimated completion is 2028-08.
What conditions does trial NCT04680065 study?
This clinical trial studies the following conditions: Multiple System Atrophy.
What interventions are being tested in trial NCT04680065?
The interventions under investigation include: AAV2-GDNF gene therapy (BIOLOGICAL), Sham (Placebo) Surgery (PROCEDURE).
Who is sponsoring clinical trial NCT04680065?
This trial is sponsored by Brain Neurotherapy Bio, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04680065 being conducted?
This trial has 7 study locations across California, Florida, Massachusetts, Michigan, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Multiple System Atrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04680065's enrollment target sits among peer trials
9 28th of 30 higher than 3 of 30 other Multiple System Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Multiple System Atrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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