View clinical trials related to Aphasia, Primary Progressive.
Filter by:Given the expansion of indications for genetic testing and our understanding of conditions for which the results change medical management, it is imperative to consider novel ways to deliver care beyond the traditional genetic counseling visit, which are both amenable to large-scale implementation and sustainable. The investigators propose an entirely new approach for the implementation of genomic medicine, supported by the leadership of Penn Medicine, investigating the use of non-geneticist clinician and patient nudges in the delivery of genomic medicine through a pragmatic randomized clinical trial, addressing NHGRI priorities. Our application is highly conceptually and technically innovative, building upon expertise and infrastructure already in place. Innovative qualities of our proposal include: 1) Cutting edge EHR infrastructure already built to support genomic medicine (e.g., partnering with multiple commercial genetic testing laboratories for direct test ordering and results reporting in the EHR); 2) Automated EHR-based direct ordering or referring by specialist clinicians (i.e., use of replicable modules that enable specialist clinicians to order genetic testing through Epic Smartsets, including all needed components, such as populated gene lists, smartphrases, genetic testing, informational websites and acknowledgement e-forms for patient signature); 3) EHR algorithms for accurate patient identification (i.e., electronic phenotype algorithms to identify eligible patients, none of which currently have phenotype algorithms present in PheKB; 4) Behavioral economics-informed implementation science methods: This trial will be the first to evaluate implementation strategies informed by behavioral economics, directed at clinicians and/or patients, for increasing the use of genetic testing; further it will be the first study in this area to test two forms of defaults as a potential local adaptation to facilitate implementation (ordering vs. referring); and 5) Dissemination: In addition to standard dissemination modalities,PheKB95, GitHub and Epic Community Library, the investigators propose to disseminate via AnVIL (NHGRI's Genomic Data Science Analysis, Visualization, and Informatics Lab-Space). Our results will represent an entirely new paradigm for the provision of genomic medicine for patients in whom the results of genetic testing change medical management.
This is a Phase 1, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of VES001 in a two part followed by a multicenter, open-label Phase 1b study in asymptomatic GRN mutation carriers. Part A will evaluate the safety, tolerability, PK, and PD of single doses of VES001 in healthy volunteers. Part B will evaluate the safety, tolerability, PK, and PD of multiple doses of VES001 in healthy volunteers.
This study is designed to evaluate the impact on the quality of life and wellbeing of a person-centered online dance program on people living with dementia or MCI and care partners. The duration of the study will be 1 year. Each participant in the study will be followed for approximately 4 months. The study includes joining a weekly 1-hour dance program online on Zoom for 12 weeks. Prior and after the dance program, participants will meet with the research coordinator to answer some questionnaires about wellbeing and reflections on their experience in the program. After the completion of the dance program, participants will be invited to join a focus group to reflect of the impact of the program with fellow participants. The study will enroll up to 72 participants. This includes 36 dyads of persons living with dementia or MCI and their care partners. The study will enroll community-dwelling people living with a diagnosis of mild cognitive impairment or mild to moderate-stage dementia and care partners living in the United States.
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate diverse disease research using cells from the body (such as skin or blood cells) to make stem cells and other types of cells, conduct research on the samples, perform genetic testing, and store the samples for future use. Through this research, researchers hope to identify future treatments or even cures for the major diseases of our time.
Insomnia is a highly common, chronic disorder that is distressful for the patient but also for caregivers and can give rise to a heavy burden on the healthcare team. Sleeping aids like benzodiazepines and other sedatives (e.g., zolpidem, zopiclone) have been widely used to help treat insomnia. However, sleeping aids are also known to cause adverse drug reactions such as drowsiness and dizziness, that increases the risk of falls, driving impairment, visual impairment, cognitive impairment, and upon discontinuation may cause paradoxical rebound insomnia, delirium, and nightmares all of which exacerbate the initial insomnia. All of the negative aspects of sleeping aid use are exaggerated for older, frail adults. Some patients experience an early (young-age) onset dementia with a substantial component of insomnia. Due to the many risks associated with traditional sleeping aids they are often inappropriate in adults living with cognitive impairment and/or frailty. Lemborexant comes from a new class of medications for insomnia. Lemborexant is a dual orexin receptor antagonist that blocks the binding of wake-promoting neuropeptides orexin A and orexin B to their receptors orexin 1 receptor (OX1R) and orexin 2 receptor (OX2R), which is thought to suppress wake drive. Unlike other traditional sleeping aids, lemborexant has not shown to be significantly associated with driving impairment, rebound insomnia, or dependence/withdrawal symptoms. Also, in clinical trials it only rarely causes the types of adverse events associated with benzodiazepines and other traditional sedatives and is less often associated with discontinuations due to adverse events. While lemborexant is available on the Canadian market it is unclear how this medication will be tolerated by patients living with an early onset dementia. Understanding the effectiveness and tolerability of lemborexant will be helpful in an N of 1 trial to understand the details of effect and effectiveness in individual patients.
The purpose of this study is to find out how the language of people with Primary Progressive Aphasia is affected by Propranolol. Propranolol is not FDA approved for the treatment of Primary Progressive Aphasia. Propranolol is FDA approved for the treatment of heart conditions such as blood pressure. This research is being done because there are currently no drug treatment options for language impairments and anxiety often experienced by people with Primary Progressive Aphasia.
The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). FTD-GRN is an early-onset form of dementia, a progressive brain disorder that affects behavior, language and movement. These symptoms result from below normal levels of a protein called progranulin (PGRN) in the brain, which leads to the death of nerve cells (neurons), affecting the brain's ability to function. The main questions that the study aims to answer are: 1. Is a one-time treatment with AVB-101 safe for patients with FTD-GRN? 2. Does a one-time treatment with AVB-101 restore PGRN levels to at least normal levels? 3. Could AVB-101 work as a treatment to slow down or stop progression of FTD-GRN? In this study there is no placebo (a dummy pill or treatment used for comparison purposes), so all participants will receive a one-time treatment of AVB-101 delivered directly to the brain, with follow-up assessments for 5 years.
Scalp to cortex distance (SCD), as a key technological parameter of brain stimulation, has been highlighted in the guidelines of non-invasive brain stimulation. However, in the context of age-related brain changes, the region-specific SCD and its impact on stimulation-induced electric field in different types of neurodegenerative diseases remain unclear.
The primary objective of this study is to enroll an observational cohort of approximately 60 patients with PSP over the course of 24 months using a multicenter study design and to follow each of them for 12 months. The secondary objective of this study is to develop a robust solution for multi-modal remote monitoring of motor symptoms and function in PSP that can be applied to other Frontotemporal lobar degeneration (FTLD) syndromes.
Dementia is the major cause of disability and dependency among older adults worldwide affecting memory, cognitive abilities and behavior, interfering with one's ability to perform daily lives activities. Although age is the strongest known risk factor for the onset of dementia, it is not a natural or inevitable consequence of aging. Dementia not only affects older people, since up to 9% of the cases appear before 65 years. The impact of dementia is highly important in financial terms also in human costs to countries, societies and individuals. Dementia is an umbrella term for several diseases, being Alzheimer's disease (AD) the most common form, contributing to 60-70% of cases. Other major forms include Lewy bodies Dementias (LBDs) and frontotemporal dementia (FTD). The role of the gastrointestinal microbiota in human brain development and function is an area of increasing interest and research. A large number of studies suggest that the gut microbiota can influence the brain, cognition and behavior of the patients, and also modulate brain plasticity, modifying brain chemistry via various mechanisms like neural, immune and endocrine Within these last two years some studies have showed differences in the microbiota of the AD patients from healthy controls. In this sense, increasing number of studies, most of them in animal models, support the notion that probiotics have significant benefit in maintaining homeostasis of the Central Nervous System. And recent studies try to replicate this finding in AD patients with controversial results. The main objective of DEM-BIOTA project is to improve the knowledge of the relationship between microbiota and dementia. DEM-BIOTA will explore the microbiota differences between dementias: AD, LBDs, that includes: Parkinson disease dementia (PDD) and Lewy Body Dementia (LBD) and FTD-behavioral variant, also in Mild Cognitive Impairment (MCI) to study the progression; in our context (Mediterranean diet and lifestyle) and characterize them in relation to neurocognitive and neuropsychiatric symptoms as well as patient functionality (dependency level). Moreover, the capacity of a probiotic compound in reverting or improving neurocognitive and neuropsychiatric symptoms and patient functionality in a sample of AD patients will be also studied.