Neurofilament Surveillance Project (NSP)
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| ClinicalTrials.gov Identifier: NCT04516499 |
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Recruitment Status :
Recruiting
First Posted : August 18, 2020
Last Update Posted : November 18, 2022
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| Condition or disease |
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| Frontotemporal Dementia Frontotemporal Lobar Degeneration FTD-GRN |
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 : | Observational |
| Estimated Enrollment : | 335 participants |
| Observational Model: | Family-Based |
| Time Perspective: | Prospective |
| Official Title: | Remote Blood Biomarker Monitoring in Frontotemporal Lobar Degeneration: Neurofilament Surveillance Project (NSP) |
| Actual Study Start Date : | September 2, 2020 |
| Estimated Primary Completion Date : | December 2025 |
| Estimated Study Completion Date : | December 2025 |
| 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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- Neurofilament Light Chain Levels [ Time Frame: 36 months ]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
- Intersubject variability of plasma NfL measurements [ Time Frame: 36 months ]Concentration of plasma neurofilament light chain protein
- Logistics measure [ Time Frame: 36 months ]Participant compliance with scheduled remote blood collection and sample processing
- Clinical and MRI correlates [ Time Frame: 36 months ]To evaluate ALLFTD collected clinical and magnetic resonance imaging (MRI) neuroimaging correlates with plasma NfL levels in asymptomatic and symptomatic f-FTLD mutation carriers
- Other biomarker evaluation [ Time Frame: 36 months ]To measure other novel blood biomarkers of neurodegeneration yet to be determined
Biospecimen Retention: Samples With DNA
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 85 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | Yes |
| Sampling Method: | Non-Probability Sample |
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.
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04516499
| Contact: Rachel Acuna-Narvaez | 6503801191 | rachel.acuna-narvaez@bluefieldproject.org |
| United States, California | |
| University of California, San Francisco | Recruiting |
| San Francisco, California, United States, 94158 | |
| Contact: Dilanaz Unal 415-502-7518 dilanaz.unal@ucsf.edu | |
| Principal Investigator: Adam Boxer, MD, PhD | |
| Principal Investigator: Howie Rosen, MD | |
| United States, Florida | |
| Mayo Clinic Florida | Recruiting |
| Jacksonville, Florida, United States, 32224 | |
| Contact: Kandise Chrestensen 904-953-4215 Chrestensen.Kandise@mayo.edu | |
| Principal Investigator: Neill Graff-Radford, MBChB | |
| United States, Maryland | |
| Johns Hopkins University School of Medicine | Recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Contact: Ann Fishman 410-502-5816 ann.fishman@jhu.edu | |
| Principal Investigator: Chiadi Onyike, MD, MHS | |
| United States, Massachusetts | |
| Massachusetts General Hospital | Recruiting |
| Charlestown, Massachusetts, United States, 02129 | |
| Contact: Erin Krahn ekrahn@mgh.harvard.edu | |
| Principal Investigator: Brad Dickerson, MD | |
| United States, Minnesota | |
| Mayo Clinic | Recruiting |
| Rochester, Minnesota, United States, 55905 | |
| Contact: Breanna Bruender 507-266-3322 bruender.breanna@mayo.edu | |
| Principal Investigator: Bradley Boeve, MD | |
| United States, Missouri | |
| Washington University in St. Louis | Recruiting |
| Saint Louis, Missouri, United States, 63110 | |
| Contact: Tina Nolte nolte.tina@wustl.edu | |
| Principal Investigator: Nupur Ghoshal, MD, PhD | |
| United States, New York | |
| Columbia University | Not yet recruiting |
| New York, New York, United States, 10032 | |
| Contact: Masood Manoochehri 212-305-5710 mm2626@cumc.columbia.edu | |
| Principal Investigator: Edward Huey, MD | |
| United States, Pennsylvania | |
| University of Pennsylvania | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Contact: Julia Kwiecinski Julia.Kwiecinski@pennmedicine.upenn.edu | |
| Principal Investigator: Lauren Massimo, PhD | |
| Principal Investigator: | Adam Boxer, MD, PhD | University of California, San Francisco | |
| Principal Investigator: | Bradley Boeve, MD | Mayo Clinic | |
| Principal Investigator: | Howie Rosen, MD | University of California, San Francisco | |
| Study Director: | Laura Mitic, PhD | The Bluefield Project to Cure Frontotemporal Dementia |
| Responsible Party: | The Bluefield Project to Cure Frontotemporal Dementia |
| ClinicalTrials.gov Identifier: | NCT04516499 |
| Other Study ID Numbers: |
NSP001 |
| First Posted: | August 18, 2020 Key Record Dates |
| Last Update Posted: | November 18, 2022 |
| Last Verified: | November 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
| Plan Description: | Please consult with ALLFTD regarding individual participant data. |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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C9orf72 GRN MAPT |
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Frontotemporal Dementia Aphasia, Primary Progressive Pick Disease of the Brain Frontotemporal Lobar Degeneration Dementia Brain Diseases Central Nervous System Diseases Nervous System Diseases Neurocognitive Disorders Mental Disorders |
TDP-43 Proteinopathies Neurodegenerative Diseases Proteostasis Deficiencies Metabolic Diseases Aphasia Speech Disorders Language Disorders Communication Disorders Neurobehavioral Manifestations Neurologic Manifestations |