Actively Recruiting
A Clinical Trial of AAV2-BDNF Gene Therapy in Early Alzheimer's Disease and Mild Cognitive Impairment
Led by Mark Tuszynski · Updated on 2025-10-21
12
Participants Needed
2
Research Sites
303 weeks
Total Duration
On this page
Sponsors
M
Mark Tuszynski
Lead Sponsor
O
Ohio State University
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a first-in-human clinical trial to test whether a protein administered into the brain continuously by gene therapy, Brain-Derived Neurotrophic Factor (BDNF), will slow or prevent cell loss in the brains of people affected by Alzheimer's disease and Mild Cognitive Impairment. The protein may also activate cells in the brain that have not yet deteriorated. Gene therapy refers to the use of a harmless virus to have brain cells make the potentially protective protein, BDNF.
CONDITIONS
Official Title
A Clinical Trial of AAV2-BDNF Gene Therapy in Early Alzheimer's Disease and Mild Cognitive Impairment
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of dementia due to Alzheimer's Disease or Mild Cognitive Impairment due to Alzheimer's Disease using recognized criteria
- Mini-Mental State Exam score between 22 and 29 depending on diagnosis
- No significant cerebral vascular disease (modified Hachinski score of 4 or less)
- Age between 50 and 80 years
- EEG without epileptiform abnormalities
- Stable permitted medications for at least one month prior to screening
- No depression indicated by Geriatric Depression Scale (score 8 or less)
- A caregiver available with frequent contact who agrees to support throughout the study
- Recent CT or MRI scan (within 24 months) without significant brain lesions or neurological disease
- Adequate vision and hearing to complete neuropsychological testing
- Good general health without other diseases expected to interfere with the study
- Normal or clinically insignificant blood tests and ECG
- Women must be post-menopausal or surgically sterile (not pregnant or lactating)
You will not qualify if you...
- Significant neurological diseases other than early Alzheimer's or MCI
- Aphasia impacting ability to complete study assessments
- Major depression or major psychiatric disorder within past two years
- Recent psychotic symptoms, agitation, or behavioral problems
- History of alcohol or substance abuse within past two years
- History of schizophrenia
- Recent suicidal ideation or behavior
- History of systemic cancer within past 18 months (except non-metastatic skin cancers)
- Significant or unstable medical illnesses affecting study compliance
- Recent use of excluded medications including certain beta-blockers, narcotics, anti-Parkinsonian drugs, neuroleptics, benzodiazepines, corticosteroids, anticonvulsants, anticoagulants
- Use of investigational drugs within 30 days prior to treatment
- Contraindications to MRI or gadolinium contrast
- History of gene therapy or investigational Alzheimer's treatments
- Investigator judgment of inability to comply with study procedures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
University of California - San Diego
La Jolla, California, United States, 92093
Actively Recruiting
2
The Ohio State University
Columbus, Ohio, United States, 43210
Actively Recruiting
Research Team
A
Andrea Davis, MS
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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