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

NCT05040217 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-10-21

No results posted yet for this study

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.

Conditions

Interventions

GENETIC

AAV2-BDNF Gene Therapy

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

BIOLOGICAL

AAV2-BDNF Gene Therapy

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

Sponsors & Collaborators

  • Ohio State University

    collaborator OTHER

  • Mark Tuszynski

    lead OTHER

Principal Investigators

  • Mark Tuszynski, M.D., Ph.D. · University of California, San Diego

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
50 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-07
Primary Completion
2027-12-01
Completion
2027-12-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05040217 on ClinicalTrials.gov