Ignite Creation Date:
2024-05-06 @ 3:05 PM
Last Modification Date:
2024-10-26 @ 1:42 PM
Study NCT ID:
NCT04516499
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-11-14
First Post:
2020-08-11
Brief Title:
Neurofilament Surveillance Project NSP
Sponsor:
The Bluefield Project to Cure Frontotemporal Dementia
Organization:
The Bluefield Project to Cure Frontotemporal Dementia
Study Overview
Official Title:
Remote Blood Biomarker Monitoring in Frontotemporal Lobar Degeneration Neurofilament Surveillance Project NSP
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-11
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 biomarker study designed to collect and analyze blood specimens from individuals carrying known familial frontotemporal lobar degeneration f-FTLD mutations compared to a control group of individuals without known f-FTLD mutations The NSP is an ancillary study to the ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration ALLFTD study NCT04363684 More information can be found at httpswwwallftdorg
Detailed Description:
Frontotemporal Lobar Degeneration FTLD a group of clinically heterogeneous neurodegenerative diseases characterized by progressive deterioration of the frontal and temporal cortices as well as basal ganglia and brainstem structures is a common cause of neurodegenerative dementia in people who are less than 60 years old at onset It is uniformly fatal FTLD is a rare disease with an estimated prevalence of approximately 5-22 per 100000 There are no approved treatments however several investigational agents are in human trials and a variety of novel agents are poised to enter human development Experience from other neurodegenerative diseases suggests that potential disease modifying treatments are most likely to be efficacious if initiated before the onset of symptoms
Approximately 25 of FTLD cases are familial f-FTLD and due to autosomal dominant mutations in one of three genes C9orf72 progranulin GRN or microtubule associated protein tau MAPT Many of the new therapies entering the clinic directly target one of these genetic causes and raise the possibility that the clinical features of FTLD could be delayed or prevented in these individuals if an efficacious therapy was initiated prior to the onset of symptoms The major barrier to determining efficacy of novel therapeutic agents for f-FTLD in such prevention trials is the lack of an endpoint that can indicate therapeutic efficacy prior to the onset of symptoms Our preliminary data strongly suggest that plasma neurofilament light chain NfL could serve as such a biomarker
This non-interventional study is in preparation for pivotal clinical trials Up to 335 participants will provide blood remotely via visits from traveling mobile research nurses four times a year for three years 4355 samples total to enable the observation of peripheral NfL levels longitudinally during disease onset and progression with the ultimate goal of qualifying plasma NfL as an endpoint for f-FTLD prevention trials The NSP study is an ancillary study to ALLFTD and biomarker data collected in the NSP will be correlated with ALLFTD clinical data More information on the NSP study may be found at httpswwwallftdorgnsp
Approximately 25 of FTLD cases are familial f-FTLD and due to autosomal dominant mutations in one of three genes C9orf72 progranulin GRN or microtubule associated protein tau MAPT Many of the new therapies entering the clinic directly target one of these genetic causes and raise the possibility that the clinical features of FTLD could be delayed or prevented in these individuals if an efficacious therapy was initiated prior to the onset of symptoms The major barrier to determining efficacy of novel therapeutic agents for f-FTLD in such prevention trials is the lack of an endpoint that can indicate therapeutic efficacy prior to the onset of symptoms Our preliminary data strongly suggest that plasma neurofilament light chain NfL could serve as such a biomarker
This non-interventional study is in preparation for pivotal clinical trials Up to 335 participants will provide blood remotely via visits from traveling mobile research nurses four times a year for three years 4355 samples total to enable the observation of peripheral NfL levels longitudinally during disease onset and progression with the ultimate goal of qualifying plasma NfL as an endpoint for f-FTLD prevention trials The NSP study is an ancillary study to ALLFTD and biomarker data collected in the NSP will be correlated with ALLFTD clinical data More information on the NSP study may be found at httpswwwallftdorgnsp
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