RecruitingPhase 1NCT05040217

A Clinical Trial of AAV2-BDNF Gene Therapy in Early Alzheimer's Disease and Mild Cognitive Impairment

A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of AAV2-BDNF [Adeno-Associated Virus (AAV)-Based, Vector-Mediated Delivery of Human Brain Derived Neurotrophic Factor] in Subjects With Early Alzheimer's Disease and Mild Cognitive Impairment


Sponsor

Mark Tuszynski

Enrollment

12 participants

Start Date

Feb 7, 2022

Study Type

INTERVENTIONAL

Conditions

Summary

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.


Eligibility

Min Age: 50 YearsMax Age: 80 Years

Plain Language Summary

Simplified for easier understanding

This trial is testing a gene therapy approach that delivers a brain-supporting protein called BDNF (Brain-Derived Neurotrophic Factor) directly into the brain of patients with early-stage Alzheimer's disease or mild cognitive impairment, using a modified harmless virus (AAV2) as a delivery vehicle. **You may be eligible if...** - You are between 50 and 80 years old with a diagnosis of mild Alzheimer's dementia (MMSE score 22–28) or mild cognitive impairment - You have no significant vascular brain disease - Your brain EEG shows no seizure activity - Your medications have been stable for at least one month - A reliable study partner (like a family member) can attend visits with you **You may NOT be eligible if...** - You have another cause of dementia (such as Lewy body, frontal dementia, or vascular dementia) - You have had a seizure, stroke, or prior brain surgery - You have serious heart, kidney, or liver disease - You have active cancer, HIV, or active hepatitis - You have a psychiatric condition (like active depression or schizophrenia) not related to Alzheimer's - You are taking blood thinners like warfarin - You are unable to have an MRI Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICAAV2-BDNF Gene Therapy

AAV2-BDNF is a genetically engineered adeno-associated virus serotype 2 (AAV-2) that expresses the human BDNF cDNA.

BIOLOGICALAAV2-BDNF Gene Therapy

Gene therapy is a biological therapy delivering the BDNF gene to the brain


Locations(2)

University of California - San Diego

La Jolla, California, United States

The Ohio State University

Columbus, Ohio, United States

View Full Details on ClinicalTrials.gov

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NCT05040217


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