This study is not yet accepting patients

GDNF Gene Therapy for Multiple System Atrophy

a study on Multiple System Atrophy

Summary

Eligibility
for people ages 35-75 (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Irene Litvan, 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 San Diego
    La Jolla California 92037 United States
  • University of California Irvine
    Irvine California 92697 United States

Lead Scientist at UCSD

  • Irene Litvan, MD
    Professor, Neurosciences. Authored (or co-authored) 329 research publications.

Details

Status
not yet accepting patients
Start Date
Completion Date
(estimated)
Sponsor
Brain Neurotherapy Bio, Inc.
ID
NCT04680065
Phase
Phase 1
Study Type
Interventional
Last Updated