Summary

Eligibility
for people ages 35-75 (full criteria)
Location
at UC Irvine
Dates
study started
completion around

Description

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.

Official Title

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

Keywords

Multiple System Atrophy, MSA, Neurotrophic factor, Growth factor, Glial cell line-derived neurotrophic factor, GDNF, AAV, Gene therapy, Shy-Drager Syndrome, Atrophy, AAV2-GDNF gene therapy

Eligibility

You can join if…

Open to people ages 35-75

  • 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

You CAN'T join if...

  • 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.

Locations

  • University of California Irvine accepting new patients
    Irvine California 92697 United States
  • The Ohio State University Medical Center in progress, not accepting new patients
    Columbus Ohio 43210 United States
  • Quest Research Institute accepting new patients
    Farmington Hills Michigan 48334 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Brain Neurotherapy Bio, Inc.
ID
NCT04680065
Phase
Phase 1 Multiple System Atrophy Research Study
Study Type
Interventional
Participants
Expecting 9 study participants
Last Updated