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

NCTID	NCT05040217 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Alzheimer's Disease, Mild Cognitive Impairment
Disease Ontology Term	DOID:10652; DOID:0080832
Compound Name	AAV2-BDNF
Sponsor	Mark Tuszynski
Funder Type	Other
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	12
Results Posted	Not Available

Target Gene/Variant	BDNF
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intraparenchymal
Drug Product Type	Viral vector
Target Tissue/Cell	Entorhinal cortex & hippocampus
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type	none
Dose 1	3E11 vg/mL
Dose 2	1E12 vg/mL
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Surgical treatment of the AD patients will be complete in December 2024, and the MCI cohort is expected be completed in 2025