Ignite Creation Date:
2024-05-06 @ 4:38 PM
Last Modification Date:
2024-10-26 @ 2:13 PM
Study NCT ID:
NCT05040217
Status:
RECRUITING
Last Update Posted:
2024-05-08
First Post:
2021-06-21
Brief Title:
A Clinical Trial of AAV2-BDNF Gene Therapy in Early Alzheimers Disease and Mild Cognitive Impairment
Sponsor:
Mark Tuszynski
Organization:
University of California San Diego
Study Overview
Official Title:
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 Alzheimers Disease and Mild Cognitive Impairment
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 Alzheimers 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
Detailed Description:
This is an open label Phase I clinical trial of AAV2-BDNF gene therapy for early Alzheimers 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 Parkinsons 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
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 Parkinsons 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None