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 |
|
|
|
Sham Comparator Placebo Surgery |
|
Recruiting Locations
Vanderbilt University Medical Center
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Brain Neurotherapy Bio, Inc.