Ignite Creation Date:
2024-05-06 @ 8:25 PM
Last Modification Date:
2024-10-26 @ 3:27 PM
Study NCT ID:
NCT06372587
Status:
RECRUITING
Last Update Posted:
2024-04-18
First Post:
2024-04-04
Brief Title:
Next-Generation alzheImerS Therapeutics
Sponsor:
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Organization:
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Study Overview
Official Title:
Leveraging Genetically Encoded engiNeered protEins foR Next-Generation alzheImerS Therapeutics
Status:
RECRUITING
Status Verified Date:
2024-04
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:
ENERGISE
Brief Summary:
Is this the right time to use next-generation approaches in Alzheimers disease AD In recent years several large clinical trials testing treatments for AD have failed putting the entire field on a reset AD drug trials have almost exclusively sought to use antibodies targeted toward misfolded amyloid and tau proteins Of note although these approaches have failed they were designed to cover both familial and sporadic forms of AD On the other hand the failure in developing new effective drugs is attributed to but not limited to the highly heterogeneous nature of AD with multiple underlying hypotheses and multifactorial pathology The idea underlying this project is based on the assumption that learning and memory disorders can arise when the connections between neurons do not change appropriately in response to experience Thus by intervening on the core mechanisms of the cellular correlate of learning and memory ie synaptic plasticity the investigators expect to preserve some of the essential brain functions in AD By overcoming the limits of traditional AD therapeutic approaches the investigators will use genetically encoded engineered proteins GEEPs which the investigators developed and tested in vitro and in murine models to control their activity in living human neurons boosting synaptic plasticity Indeed outstanding and relevant progress in understanding synaptic physiology empowers the possibility to prevent or limit brain disease like never before The investigators designed GEEPs to address some of the leading causes of synaptic plasticity failures documented in AD Thus GEEPs will be tested in human induced pluripotent stem cells hiPSCs-derived living neurons obtained from reprogrammed peripheral tissues of participants with Alzheimers diseases hiPSCs will be obtained from fibroblast-derived from a skin biopsy of participants with AD and controls performed in local anesthesia using a 4 mm punch The findings will provide the first preclinical study on the effect of genetically engineered proteins to control essential pathways implicated in synaptic plasticity on AD-related cognitive decline
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None