🏛️ Verified NIH Record • Updated 2026-01-28
GDNF Gene Therapy for Multiple System Atrophy (NCT04680065)
Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
🔵 Active, Not Recruiting Phase1 💊 Investigational Drug / Biologic Sponsor: Brain Neurotherapy Bio, Inc.
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of California Irvine — Irvine, California
Age range: 35 Years to 75 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (7 Sites)
- 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
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Multiple System Atrophy
- Randomized Double-Blind Placebo-Controlled Adaptive Design Trial Of Intrathecally Administered Autologous Mesenchymal Stem Cells In Multiple System Atrophy — Rochester, Minnesota
- A Phase 2 Study of YA-101 in Patients With Multiple System Atrophy — Los Angeles, California
- A Study of Lu AF82422 in Participants With Multiple System Atrophy — Fountain Valley, California
- A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA) — Scottsdale, Arizona
- Reduction in Splanchnic Capacitance Contributes to Sympathetically Dependent Hypertension in Autonomic — Nashville, Tennessee
Explore by condition: All Multiple System Atrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.