GDNF Gene Therapy for Multiple System Atrophy

a study on Multiple System Atrophy

Summary

Eligibility
for people ages 35-75 (full criteria)
Location
at Irvine, California and other locations
Dates
study started
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 adults 35-75 years of age (inclusive)
  • Diagnosed with MSA with sporadic, adult-onset (>30 yo) with predominant parkinsonian symptoms
  • Less than 4 years from clinical diagnosis of MSA with expected survival > 3 years
  • Stable medication regimen
  • Ability to walk with or without an assistive device

You CAN'T join if...

  • Presence of idiopathic Parkinson's disease or other neurological diseases
  • Myocardial sympathetic denervation inconsistent with an MSA diagnosis
  • Presence of dementia, psychosis, substance abuse or poorly controlled depression
  • Prior brain surgery (i.e. deep brain stimulator) or other brain imaging abnormalities
  • Receiving an investigational drug
  • History of cancer or poorly controlled medical conditions that would increase surgical risk

  • Inability to tolerate laying flat in an MRI 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
Contact us about this trial