Clinical Trials

The purpose of this study is to evaluate sporadic (s-) and familial (f-) frontotemporal lobar degeneration (FTLD) patients and asymptomatic family members of f-FTLD patients, characterizing the cohorts longitudinally and informing clinical trial design.  FTLD is a neurodegenerative disorder of the nervous system which there are no approved treatments or cures.

The study has two arms: a “longitudinal arm” involving a comprehensive assessment of clinical, functional, imaging, and biofluid data collection, and a “biofluid-focused arm” involving limited clinical data to accompany biospecimen collection.

The purpose of this study is 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 primary purpose of this study is to evaluate the efficacy of AL001 compared with placebo in carriers of progranulin gene (GRN) mutations causative of frontotemporal dementia (FTD) as measured by the Clinical Dementia Rating Dementia Staging Instrument PLUS National Alzheimer’s Disease Coordinating Center frontotemporal lobar degeneration Behavior & Language Domains Sum of Boxes (CDR® plus NACC FTLD-SB).

The purposes of this study are to investigate the safety, tolerability, and pharmacodynamics of FRM-0334 in subjects with prodromal to moderate frontotemporal dementia with granulin mutation.

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of AL001 in participants with a  frontotemporal dementia.

The objectives of the study are to; (1) estimate the change in disease -related cognitive decline over 1 year on a battery of cognitive tests administered to participants with early-stage symptomatic Behavioral Variant Frontotemporal Dementia (bvFTD) phenotypic variant; (2) identify the cognitive test or brief battery of cognitive tests which are the most sensitive to detect bvFTD progression; (3) determine the optimal schedule of administration of cognitive tests to detect bvFTD progression; (4) evaluate the relationship between cognitive tests and measures of behavior, function, caregiver's burden, quality of life (QOL); and (5) obtain blood samples for genetic and exploratory biomarkers ...

This study is being done to learn more about normal thinking and behavior, mild thinking and behavior problems, Frontotemporal Dementia and other forms of dementia in families in which one or more relatives have a mutation associated with Frontotemporal Dementia.

The purpose of this study is to demonstrate the safety and efficacy of TRx0237 in the treatment of patients with behavioral variant frontotemporal dementia (bvFTD).

The purpose of this study is to provide subjects who have completed participation in a Phase 2 or Phase 3 trial with TRx0237 continued access to therapy and to evaluate the long-term safety of TRx0237.

The purpose of this study is to collect blood samples for DNA analysis from patients clinically diagnosed with Alzheimers disease, Lewy Body disease, and Frontotemporal degeneration.

The primary objective of this study is to assess the safety and tolerability of TPN-101 in patients with C9ORF72 amyotrophic lateral sclerosis (ALS)/frontotemporal dementia (FTD).

This study is intended to obtain clinical information and establish a repository of DNA, RNA, peripheral blood monocyte, lymphocyte and skin tissue samples from people with ALS and related neurodegenerative motor neuron diseases, people with a family history of these conditions, and healthy people with no family history of these disorders. The samples will be used in future research to learn about how these disorders affect people, what causes these conditions, and how the investigators can tell when someone has this kind of disease. Future research may also include the generation of stem cells from stored blood cell and skin ...

The purpose of this study is to develop and test the effectiveness of an investigational imaging technique called magnetic resonance elastography (MRE) to measure the stiffness (mechanical properties) of tissues.

The purpose of this study is to collect clinical data, serial blood samples for plasma, serum, DNA and RNA extraction, and a single time blood sample for peripheral blood monocytes for the preparation of stem cell lines and lymphocytes for the preparation of lymphoblastoid cell lines. There will also be a single time skin biopsy sample for tissue examination and preparation of skin fibroblasts from patients who have familial or sporadic ALS, ALS-FTD or similar neurodegenerative motor neuron disorders, and are participating in NINDS funded research projects in the Mayo Clinic ALS Center.

The purpose of this study is to collect CSF and blood samples that can be used in future research studies to identify potential biomarkers in blood and CSF collected in ALS patients. Biomarkers are non-genetic elements in your blood and CSF that may help us diagnose and monitor ALS more easily. There are no readily available sources of longitudinal CSF, plasma or serum samples from people with ALS, ALS-FTD or similar neurodegenerative disorders or their family members for use in the identification of potential ALS biomarkers. Future research will examine potential biomarkers in blood and CSF collected over time to ...

The purpose of this study is to optimize profile questionnaire and matching algorithm developed in Phase I and implement in final website design, and to determine if algorithmically matched participants have statistically significant increase in match satisfaction and self-reported sense of resiliency and quality of life over randomly matched caregivers.

This study is being done to learn more about normal memory and aging, mild memory and thinking problems, Alzheimer's disease and other forms of dementia.

The purpose of this study is to collect and study blood samples that can be used in current and future research studies to identify genetic risk factors in ALS and identify potential biomarkers in blood collected in ALS patients. Biomarkers are non-genetic elements in your blood that may help us diagnose and monitor ALS more easily. There are no readily available sources of longitudinal DNA, RNA, monocytes, serum or plasma from people with ALS, ALS-FTD or similar neurodegenerative disorders or their family members for use in the identification of potential changes in gene structure over time or biomarkers in ALS. ...

The purpose of this study is to facilitate focus groups to assess/identify important qualities and characteristics that dementia caregivers are looking for in a supportive person, and to design a prototype website for matching current and former caregivers.

Frontotemporal Lobar Degeneration (FTLD) is the neuropathological term for a collection of rare neurodegenerative diseases that correspond to four main overlapping clinical syndromes: frontotemporal dementia (FTD), primary progressive aphasia (PPA), corticobasal degeneration syndrome (CBS) and progressive supranuclear palsy syndrome (PSPS). The goal of this study is to build a FTLD clinical research consortium to support the development of FTLD therapies for new clinical trials. The consortium, referred to as Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL), will be headquartered at UCSF and will partner with six patient advocacy groups to manage the consortium. Participants will be evaluated at ...

This study is being done to learn more about normal memory and aging, mild memory and thinking problems, Alzheimer's disease and other forms of dementia. This study will help us determine how often memory problems occur in people in our community, and help to identify factors that may influence changes in memory and thinking skills.

The goal of this study is to gain a better understanding of the status of advanced care planning among caregivers of patients with dementia and examine how this differs by race and disease stage.

To further investigate biomarkers in CSF as possible predictors for mild cognitive impairment and dementia