GDNF Gene Therapy for Multiple System Atrophy

January 28, 2026 updated by: Brain Neurotherapy Bio, Inc.

Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in 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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

9

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Irvine, California, United States, 92697
        • University of California Irvine
    • Florida
      • Boca Raton, Florida, United States, 33486
        • Parkinson's Disease and Movement Disorders Center of Boca Raton
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Michigan
      • Farmington Hills, Michigan, United States, 48334
        • Quest Research Institute
    • New York
      • New York, New York, United States, 10016
        • NYU Langone Health
    • Ohio
      • Columbus, Ohio, United States, 43210
        • The Ohio State University Medical Center
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

35 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male and female 35-75 years of age (inclusive)
  • Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and > 30 years of age
  • Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years
  • Stable anti-parkinsonian medication regimen
  • Ability to walk a distance of 25 feet with or without an assistive device

Exclusion Criteria:

  • Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases
  • Presence of dementia, psychosis, substance abuse or poorly controlled depression
  • Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities
  • History of cancer or poorly controlled medical conditions that would increase surgical risk
  • Received investigational agent within 12 weeks
  • Inability to tolerate laying flat in an MRI and/or allergy to gadolinium

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Active Treatment
Biological: AAV2-GDNF gene therapy
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Sham Comparator: Placebo Surgery
Procedure: Sham (Placebo) Surgery
Bilateral partial burr/twist holes without dural penetration

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations
Time Frame: 3 years
Number of TEAE and SAE's reported post-treatment.
3 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)
Time Frame: 12 months
Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) and compared to placebo. UMSARS total scores range from 0-104 points with higher scores indicating greater severity of impairment.
12 months
Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane
Time Frame: 12 months
Percent and absolute change in ratio of specific to non-specific binding of 123I FP-CIT to DaT from baseline and compared to placebo by Single Photon Emission Computed Tomography (SPECT) dopamine transporter (DaT) imaging
12 months
Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)
Time Frame: 12 months
Change from baseline and compared to placebo in the Multiple System Atrophy Quality of Life (MSA-QoL) scale. MSA-QoL is a self-reported questionnaire that measures MSA impact in day to day activities. Scale consists of 40 items with a five response option format (0 - no problem to 4 extreme problem) and a "not applicable" response option.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Brain Neurotherapy Bio, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 3, 2023

Primary Completion (Estimated)

August 1, 2026

Study Completion (Estimated)

August 1, 2028

Study Registration Dates

First Submitted

December 15, 2020

First Submitted That Met QC Criteria

December 20, 2020

First Posted (Actual)

December 22, 2020

Study Record Updates

Last Update Posted (Actual)

January 30, 2026

Last Update Submitted That Met QC Criteria

January 28, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MSA-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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