- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04516499
Neurofilament Surveillance Project (NSP)
Remote Blood Biomarker Monitoring in Frontotemporal Lobar Degeneration: Neurofilament Surveillance Project (NSP)
Study Overview
Status
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 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.
Study Type
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Rachel Acuna-Narvaez
- Phone Number: 6503801191
- Email: rachel.acuna-narvaez@bluefieldproject.org
Study Locations
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California
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San Francisco, California, United States, 94158
- University of California, San Francisco
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Florida
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Jacksonville, Florida, United States, 32224
- Mayo Clinic Florida
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Maryland
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Baltimore, Maryland, United States, 21287
- Johns Hopkins University School of Medicine
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Massachusetts
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Charlestown, Massachusetts, United States, 02129
- Massachusetts General Hospital
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Minnesota
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Rochester, Minnesota, United States, 55905
- Mayo Clinic
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Missouri
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Saint Louis, Missouri, United States, 63110
- Washington University in St. Louis
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New York
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New York, New York, United States, 10032
- Columbia University
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- University of Pennsylvania
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Sampling Method
Study Population
This study is an ancillary study to ALLFTD. Thus, all NSP participants will be recruited directly from the longitudinal arm of ALLFTD.
Roughly equivalent numbers of participants will be enrolled from each of the following groups:
- Members of families with known mutations in C9orf72
- Members of families with known mutations in GRN
- Members of families with known mutations in MAPT
Although participant must be from a family with a known mutation, the participant themselves need not know their personal mutation status.
Description
Inclusion Criteria:
- Male or female
- Ages 18-85
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Is enrolled in the longitudinal arm of ALLFTD
- Is a member of a family with a known mutation in C9orf72, GRN or MAPT
Exclusion Criteria:
- Any permanent contra-indication to repeated blood draws, such as poor venous access.
- Any conditions or circumstances which, in the opinion of the investigator, would not allow participation in the study.
Study Plan
How is the study designed?
Design Details
- Observational Models: Family-Based
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
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f-FTLD mutation carriers
All participants must be from a family with f-FTLD mutations.
The f-FTLD mutation carrier group members will have their genetic status tested and included in this group if a f-FTLD mutation is observed.
Participants do not need to know or be told their genetic status.
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Non-mutation carriers from families with f-FTLD mutations
All participants must be from a family with f-FTLD mutations.
The non-mutation carrier group members will have their genetic status tested and included in this group if they do not have a f-FTLD mutation.
Participants do not need to know or be told their genetic status.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Neurofilament Light Chain Levels
Time Frame: 36 months
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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
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36 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Intersubject variability of plasma NfL measurements
Time Frame: 36 months
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Concentration of plasma neurofilament light chain protein
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36 months
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Logistics measure
Time Frame: 36 months
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Participant compliance with scheduled remote blood collection and sample processing
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36 months
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Clinical and MRI correlates
Time Frame: 36 months
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To evaluate ALLFTD collected clinical and magnetic resonance imaging (MRI) neuroimaging correlates with plasma NfL levels in asymptomatic and symptomatic f-FTLD mutation carriers
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36 months
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Other biomarker evaluation
Time Frame: 36 months
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To measure other novel blood biomarkers of neurodegeneration yet to be determined
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36 months
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Collaborators and Investigators
Investigators
- Principal Investigator: Adam Boxer, MD, PhD, University of California, San Francisco
- Principal Investigator: Howie Rosen, MD, University of California, San Francisco
- Study Director: Laura Mitic, PhD, The Bluefield Project to Cure Frontotemporal Dementia
- Principal Investigator: Bradley Boeve, MD, Mayo Clinic
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Mental Disorders
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Neurocognitive Disorders
- Neurodegenerative Diseases
- TDP-43 Proteinopathies
- Proteostasis Deficiencies
- Dementia
- Language Disorders
- Communication Disorders
- Speech Disorders
- Aphasia
- Frontotemporal Dementia
- Aphasia, Primary Progressive
- Pick Disease of the Brain
- Frontotemporal Lobar Degeneration
Other Study ID Numbers
- NSP001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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