Clinical Trials Logo

Clinical Trial 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.


Clinical Trial Description

This is an open label Phase I clinical trial of AAV2-BDNF gene therapy for early Alzheimer's Disease (AD) and Mild Cognitive Impairment (MCI) in 12 participants. BDNF is a nervous system growth factor that regulates neuronal function in key memory circuits of the brain (the entorhinal cortex and hippocampus). BDNF reduces cell loss, stimulates cell function, and builds new connections (synapses) between brain cells in animal models. This clinical trial will use techniques of gene therapy because the candidate therapeutic protein, BDNF, does not cross the blood brain barrier (BBB). Two previous clinical programs of Nerve Growth Factor (NGF) gene therapy for AD and Neurturin gene therapy for Parkinson's disease in over 120 patients provided evidence that degenerating neurons respond to growth factors with classic "trophic" responses in the human brain. Participants will undergo one gene transfer procedure. Thus, dosing is performed only once, and repeat dosing or daily medications are not expected to be required. 12 participants will be enrolled in this Phase I trial, 6 with early AD and 6 with MCI. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05040217
Study type Interventional
Source University of California, San Diego
Contact Lori Graham
Phone 858-822-3372
Email [email protected]
Status Recruiting
Phase Phase 1
Start date March 1, 2021
Completion date March 1, 2025

See also
  Status Clinical Trial Phase
Completed NCT05040048 - Taxonomy of Neurodegenerative Diseases : Observational Study in Alzheimer's Disease and Parkinson's Disease
Withdrawn NCT03316898 - A FDG-PET Study of AGN-242071 Added to Standard-of-Care (Donepezil ± Memantine) for the Treatment of Participants With Mild to Moderate Alzheimer's Disease Phase 1
Withdrawn NCT02860065 - CPC-201 Alzheimer's Disease Type Dementia: PET Study Phase 2
Active, not recruiting NCT01315639 - New Biomarker for Alzheimer's Disease Diagnostic N/A
Not yet recruiting NCT03740178 - Multiple Dose Trial of MK-4334 in Participants With Alzheimer's Clinical Syndrome (MK-4334-005) Phase 1
Terminated NCT03307993 - Positron Emission Tomography (PET) Study in Patients With Alzheimer's Disease Phase 1
Not yet recruiting NCT05048966 - Comparison of Two Group Wellness Interventions for Individuals With Neurologic Conditions and Their Support Persons N/A
Recruiting NCT02912936 - A Medium Chain Triglyceride Intervention for Patients With Alzheimer Disease N/A
Recruiting NCT02899091 - Evaluation of the Safety and Potential Therapeutic Effects After Intravenous Transplantation of CB-AC-02 in Patients With Alzheimer's Disease Phase 1/Phase 2
Completed NCT02907567 - Clinical Trial of CT1812 in Mild to Moderate Alzheimer's Disease Phase 1/Phase 2
Not yet recruiting NCT02868905 - Identification of Epigenetic Markers Common to Obesity and Alzheimer's Disease in Women N/A
Completed NCT02516046 - 18F-AV-1451 Autopsy Study Phase 3
Completed NCT02580305 - SUVN-502 With Donepezil and Memantine for the Treatment of Moderate Alzheimer's Disease- Phase 2a Study Phase 2
Terminated NCT02521558 - Effectiveness of Home-based Electronic Cognitive Therapy in Alzheimer's Disease N/A
Recruiting NCT02247180 - Cognitive Rehabilitation in Alzheimer`s Disease N/A
Completed NCT02260167 - Treatment of Alzheimer's and Dementia With the Metabolism, Infections, Nutrition, Drug Elimination (MIND) Protocol N/A
Terminated NCT02220738 - Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ABT-957 in Subjects With Mild-to-Moderate Alzheimer's Disease on Stable Doses of Acetylcholinesterase Inhibitors Phase 1
Completed NCT02256306 - The PLasma for Alzheimer SymptoM Amelioration (PLASMA) Study N/A
Completed NCT02317523 - Alzheimer's Caregiver Coping: Mental and Physical Health N/A
Completed NCT01826110 - PET Imaging Study Using [11C]PIB in Subjects With AD & Healthy Volunteers Phase 1