GDNF Gene Therapy for Multiple System Atrophy

Description

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

Multiple System Atrophy

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Study Overview

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Study Details

Study overview

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.

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

GDNF Gene Therapy for Multiple System Atrophy

Condition
Multiple System Atrophy
Intervention / Treatment

-

Contacts and Locations

Irvine

University of California Irvine, Irvine, California, United States, 92697

Boca Raton

Parkinson's Disease and Movement Disorders Center of Boca Raton, Boca Raton, Florida, United States, 33486

Boston

Massachusetts General Hospital, Boston, Massachusetts, United States, 02114

Farmington Hills

Quest Research Institute, Farmington Hills, Michigan, United States, 48334

New York

NYU Langone Health, New York, New York, United States, 10016

Columbus

The Ohio State University Medical Center, Columbus, Ohio, United States, 43210

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

35 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Brain Neurotherapy Bio, Inc.,

Study Record Dates

2028-12