View clinical trials related to Aphasia, Primary Progressive.
Filter by:Primary progressive aphasias (PPA) represent a challenging group of degenerative language diseases that has led to growing interest in the scientific and medical community. However, a full-blown cognitive/linguistic, anatomic and biologic characterization of the three main variants remains incomplete given that the available data derive from relatively small patient samples. Such a three-fold characterisation will be an major milestone with the prospective of providing the rationale for therapeutic interventions comprising specific rehabilitations protocols and pharmacological trials. The present study addresses theses issues in the three PPA main variants through a cross-sectional and longitudinal investigation exploring 1) cognitive/linguistic features, 2) anatomic/metabolic specifications (MRI-VBM, MRI-fiber tracking, functional connectivity - MRI resting state, PET), and 3) biologic aspects (CSF biomarkers, genetic screening).
The purpose of this study is to provide subjects who have completed participation in a Phase 2 or Phase 3 trial of LMTM continued access to therapy and to evaluate the long-term safety of LMTM.
Frontal patients are impaired in categorisation and analogical reasoning tasks, and different functional imaging studies from our group have shown the involvement of the prefrontal cortex in categorisation and analogy tasks. The aim of this project is to test our hypotheses about the role of the prefrontal cortex in explicit and implicit categorisation and analogy tasks.
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.
Niemann-Pick disease type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence. The causative genes NPC1 (about 95% of cases) and NPC2 (about 5% of cases) are involved in the intracellular trafficking of lipids and cholesterol. Mutations on either of these genes lead to progressive accumulation of unesterified cholesterol and other lipids in the central nervous system (CNS). Vorinostat is a histone deacetylase inhibitor that has been shown in vivo to increase mutant NPC1 protein levels and to reverse cellular accumulation of unesterified cholesterol. Vorinostat has been labeled by the FDA for treatment of cutaneous T-cell lymphoma. In this Phase I, non-randomized, open-label, single-center study, we plan to study whether Vorinostat can be repurposed to treat patients with NPC1. Our primary objective is to determine the safety and tolerability of Vorinostat in NPC1 disease. Our secondary objectives will be to determine biochemical efficacy of Vorinostat to increase expression of NPC1 protein and normalize lipid and protein biomarkers. This study will enroll up to 12 NPC1 patients and test the safety of two dose levels (200 and 400 mg). Drug will be administered on a 3 days on/4 days off schedule for 3 months at each dose level. Patients will be evaluated at the NIH Clinical Center at 0, 3 and 6 months. Safety will be assessed by adverse events (AEs), clinical laboratory tests and physical examinations. Biochemical efficacy will be assessed by measurement of serum and cerebral spinal fluid biomarkers. Clinical efficacy will be evaluated by audiologic testing, assessment ataxia, and swallowing studies.
Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US. Frequently, patients show progressive worsening of symptoms for several years before they get diagnosed. Since many of these diseases can be managed therapeutically, it is important to identify and treat patients in order to avoid organ damage. The investigators aim to undertake a screening study that identifies undiagnosed patients with lysosomal storage disorders and determine the prevalence of these diseases with special focus on underrepresented minority groups.
The goal of this study is to assess [18F]MNI-777 PET imaging as a tool to detect tau pathology in the brain of individuals who carry a clinical diagnosis of a tauopathy, including: Alzheimer's Disease (AD),Parkinson's disease (PD) Progressive Supranuclear Palsy (PSP), chronic traumatic encephalopathy (CTE) and Frontal Temporal Dementia (FTD) and age- and gender-matched healthy subjects.
People diagnosed with young onset dementia are today mostly assigned to the same healthcare services as people developing dementia at an older age. They and their families are however in a quite different life situation, which is likely to generate different challenges and specific needs for tailored healthcare services, of importance in maintaining their perceived quality of life. The investigators of this study wish to assess the factors influencing these families' quality of life, their specific needs and their use of healthcare services by the use a combination of quantitative and qualitative methods. The main aim of this study is to provide better future healthcare services to these families, and to develop a programme for optimal collaboration between specialist healthcare services and the local dementia teams.
The Virtual Physiological Human: DementiA Research Enabled by IT (VPH-DARE@IT) is a four-year IT-project funded through the European Union (EU). The project consortium involves a total of 21 universities and industrial partners from 10 European countries. The project delivers the first patient-specific predictive models for early differential diagnosis of dementia and their evolution. An integrated clinical decision support platform will be validated / tested by access to a dozen databases of international cross-sectional and longitudinal studies. As a part of the VPH-DARE@IT project, a new prospective cohort will be collected in Kuopio. This prospective cohort will be used to test further the modeling approaches and tools developed by using the retrospective databases.
This study will evaluate the effects on emotions and neural activity of a one time dose of intranasal oxytocin vs. placebo in patients with FTD and healthy controls.