RecruitingAlzheimer's DiseaseMild Cognitive Impairment
A Clinical Trial of AAV2-BDNF Gene Therapy in Early Alzheimer's Disease and Mild Cognitive Impairment
Eligible age
50–80 yrs
Accepts
All genders
Locations
2 states
Healthy volunteers
No
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About this study
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.
Sponsor: Mark Tuszynski
You may qualify if…
- ✓ Specific inclusion criteria will be as follows for patients 1-6 (Mild AD dementia):
- ✓ 1. Diagnosis of dementia due to Alzheimer's Disease (AD) by National Institute of Aging (NIA) - Alzheimer's Association (AA) criteria for AD 15. The diagnosis of probable AD according to NIA criteria 15 is internationally recognized as the "gold standard" for diagnosing AD.
- ✓ 2. Mini-Mental State Exam score between 22 and 28 (inclusive).
- ✓ 3. No significant cerebral vascular disease: modified Hachinski score of ≤ 4.
- ✓ 4. Age 50 - 80 years old.
- ✓ 5. EEG is free of epileptiform abnormalities.
- ✓ 6. Permitted medications stable for at least one month prior to screening. In particular:
- ✓ 1. Subjects taking stable doses of antidepressants lacking significant anti-cholinergic side effects are acceptable (if they are not currently depressed and do not have a history of major depression within the past two years).
You may not qualify if…
- ✕ The below Exclusion Criteria apply to both the AD and MCI groups.
- ✕ 1. Any significant neurological disease other than suspected incipient disease; i.e., seizure disorder, Parkinson's disease, multi-infarct dementia, Huntington's disease, normal pressure hydrocephalus, brain tumor, progressive supranuclear palsy, subdural hematoma, multiple sclerosis, arteriovenous malformation or history of significant head trauma followed by persistent neurologic deficits or known structural abnormalities.
- ✕ 2. Individual has symptoms of aphasia which would make administration of study assessments and collection of information during study challenging.
- ✕ 3. Major depression or another major psychiatric disorder as described in DMS-IV within the past two years.
- ✕ 4. Psychotic features, agitation or behavioral problems within the last three months which could lead to difficulty in cooperating with study.
- ✕ 5. History of alcohol or substance abuse or dependence within the past two years (DMS-IV criteria).
- ✕ 6. History of schizophrenia (DMS-IV criteria).
- ✕ 7. Affirms suicidal ideation in response to questions number 4 or 5 in the C-SSRS during the past 3 months (i.e., "active suicidal ideation with some intent to act, without specific plan" or "active suicidal ideation with specific plan and intent") or affirms any of the questions contained in the Suicidal Behavior section of the C-SSRS as applicable during the past 12 months.
Where it's recruiting
California
La Jolla
Ohio
Columbus
Source: ClinicalTrials.gov · NCT05040217 · last updated 2025-10-21