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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04516499
Other study ID # NSP001
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date September 2, 2020
Est. completion date December 2026

Study information

Verified date November 2023
Source The Bluefield Project to Cure Frontotemporal Dementia
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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 https://www.allftd.org/.


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 100,000. 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 https://www.allftd.org/nsp.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 335
Est. completion date December 2026
Est. primary completion date December 2026
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 18 Years to 85 Years
Eligibility Inclusion Criteria: 1. Male or female 2. Ages 18-85 3. Provision of signed and dated informed consent form 4. Stated willingness to comply with all study procedures and availability for the duration of the study 5. Is enrolled in the longitudinal arm of ALLFTD 6. Is a member of a family with a known mutation in C9orf72, GRN or MAPT Exclusion Criteria: 1. Any permanent contra-indication to repeated blood draws, such as poor venous access. 2. Any conditions or circumstances which, in the opinion of the investigator, would not allow participation in the study.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
United States Johns Hopkins University School of Medicine Baltimore Maryland
United States Massachusetts General Hospital Charlestown Massachusetts
United States Mayo Clinic Florida Jacksonville Florida
United States Columbia University New York New York
United States University of Pennsylvania Philadelphia Pennsylvania
United States Mayo Clinic Rochester Minnesota
United States Washington University in St. Louis Saint Louis Missouri
United States University of California, San Francisco San Francisco California

Sponsors (3)

Lead Sponsor Collaborator
The Bluefield Project to Cure Frontotemporal Dementia Mayo Clinic, University of California, San Francisco

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Other Clinical and MRI correlates To evaluate ALLFTD collected clinical and magnetic resonance imaging (MRI) neuroimaging correlates with plasma NfL levels in asymptomatic and symptomatic f-FTLD mutation carriers 36 months
Other Other biomarker evaluation To measure other novel blood biomarkers of neurodegeneration yet to be determined 36 months
Primary Neurofilament Light Chain Levels To determine the longitudinal stability of plasma neurofilament light chain (NfL) measured every 3 months for 36 months in individuals at-risk for symptomatic FTLD 36 months
Secondary Intersubject variability of plasma NfL measurements Concentration of plasma neurofilament light chain protein 36 months
Secondary Logistics measure Participant compliance with scheduled remote blood collection and sample processing 36 months
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