Vanderbilt Clinical Trials

GDNF Gene Therapy for Multiple System Atrophy

Purpose

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.

Condition

Multiple System Atrophy

Eligibility

Eligible Ages: Between 35 Years and 75 Years
Eligible Genders: All
Accepts Healthy Volunteers: No

Inclusion Criteria

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

Exclusion Criteria

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.

Study Design

Phase: Phase 1
Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Up to 9 study participants meeting eligibility criteria will be randomized in a 2:1 fashion to receive either the investigational medicinal product or sham surgery in this Phase 1 trial.
Primary Purpose: Treatment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Active Treatment		Biological: AAV2-GDNF gene therapy Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Sham Comparator Placebo Surgery		Procedure: Sham (Placebo) Surgery Bilateral partial burr/twist holes without dural penetration

Recruiting Locations

Vanderbilt University Medical Center
Nashville, Tennessee 37232

Contact:
Kelly Harper
kelly.a.harper@vumc.org

More Details

Status: Recruiting
Sponsor: Brain Neurotherapy Bio, Inc.

Study Contact

Akash Pandhare, MD
336-265-2790
askfirst@askbio.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.