Summary

Eligibility
for people ages 35-75 (full criteria)
Location
at Irvine, California and other locations
Dates
study start
estimated completion
Principal Investigator
by Nicolas Phielipp, MD

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
  • Quest Research Institute accepting new patients
    Farmington Hills Michigan 48334 United States

Lead Scientist at UC Irvine

  • Nicolas Phielipp, MD
    Associate Clinical Professor, Neurology, School of Medicine. Authored (or co-authored) 11 research publications

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