ACTIVE_NOT_RECRUITING

GDNF Gene Therapy for Multiple System Atrophy

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2023-10-03
Study Completion:2028-08
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04680065

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.

Ages Eligible for Study:35 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male and female 35-75 years of age (inclusive)
  2. * Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and \> 30 years of age
  3. * Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years
  4. * Stable anti-parkinsonian medication regimen
  5. * Ability to walk a distance of 25 feet with or without an assistive device
  1. * Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases
  2. * Presence of dementia, psychosis, substance abuse or poorly controlled depression
  3. * Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities
  4. * History of cancer or poorly controlled medical conditions that would increase surgical risk
  5. * Received investigational agent within 12 weeks
  6. * Inability to tolerate laying flat in an MRI and/or allergy to gadolinium

Contacts and Locations

Study Locations (Sites)

University of California Irvine
Irvine, California, 92697
United States
Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, 33486
United States
Massachusetts General Hospital
Boston, Massachusetts, 02114
United States
Quest Research Institute
Farmington Hills, Michigan, 48334
United States
NYU Langone Health
New York, New York, 10016
United States
The Ohio State University Medical Center
Columbus, Ohio, 43210
United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
United States

Collaborators and Investigators

Sponsor: Brain Neurotherapy Bio, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-10-03
Study Completion Date2028-08

Study Record Updates

Study Start Date2023-10-03
Study Completion Date2028-08

Terms related to this study

Keywords Provided by Researchers

  • MSA
  • Multiple System Atrophy
  • Neurotrophic factor
  • Growth factor
  • Glial cell line-derived neurotrophic factor
  • GDNF
  • AAV
  • Gene therapy

Additional Relevant MeSH Terms

  • Multiple System Atrophy