View clinical trials related to Pick Disease of the Brain.
Filter by:Oxytocin is a hormone produced by the brain that appears to have important roles in social cognition and emotion in humans. In a pilot study, the effects of a single dose of oxytocin on measures of emotion recognition and behaviour in patients with Frontotemporal Dementia were investigated. The results from the pilot study suggested that oxytocin may be associated with a modest improvement in neuropsychiatric behaviours seen in patients with Frontotemporal Dementia. To further examine the safety and tolerability of oxytocin in this disorder, the present study will examine the safety and tolerability of three different doses of intranasal oxytocin administered to patients with Frontotemporal Dementia twice daily for 1 week.
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
The investigators are researching families with inherited inclusion body myopathy (IBM) and/or Paget disease of bone (PDB) and/or dementia (FTD) which is also called IBMPFD. IBMPFD is caused by mutations in the VCP gene. Our main goal is to understand how changes in the VCP gene cause the muscle, bone and cognitive problems associated with the disease. The investigators are collecting biological specimen such as blood and urine samples, family and medical histories, questionnaire data of patients with a personal or family history of VCP associated disease. Participants do not need to have all symptoms listed above in order to qualify. A select group of participants may be invited to travel to University of California, Irvine for a two day program of local procedures such as an MRI and bone scan. Samples are coded to maintain confidentiality. Travel is not necessary except for families invited for additional testing.
Development of a new MS-based biomarker for the early and sensitive diagnosis of Niemann Pick Type C disease from Blood (plasma)
This study is designed to document the loss of sociomoral emotions (like empathy, guilt, and embarrassment) in patients with behavioral variant frontotemporal dementia. The loss of these emotions, which function as the motivators for social behavior, will manifest in specific interpersonal behaviors. These behaviors will correlate with regional changes in regional changes in medial frontal and anterior temporal lobes. These social and emotional changes will be compared with a young-onset Alzheimer's disease comparison group.
The primary objective of the study is to obtain preliminary safety and tolerability data with davunetide (NAP, AL-108) in patients with a tauopathy (frontotemporal lobar degeneration [FTLD] with predicted tau pathology, corticobasal degeneration syndrome [CBS] or progressive supranuclear palsy [PSP]). The secondary objectives of this study are to obtain preliminary data on short term changes (at 12 weeks) in a variety of clinical, functional and biomarker measurements from baseline, including cerebrospinal fluid (CSF) tau levels, eye movements, and brain MRI measurements.
Investigations into the components of cognition damaged in frontotemporal dementia (FTD) demonstrate that patients with FTD show deficits in facial and verbal expression recognition, lack insight into what others think or might do (theory of mind skills), and in decision making tasks requiring processing of positive versus negative feedback. These cognitive functions are thought to be critical for appropriate social behavioural regulation (Blair, 2003). Recent studies in animal models and humans suggest that the neuropeptide oxytocin is an important mediator of social behavior and that oxytocin may facilitate emotion recognition, theory of mind processing, and prosocial behaviors (Donaldson and Young, 2008). Together, these findings suggest that upregulation of oxytocin dependent mechanisms of social and emotional cognition may be a valuable treatment approach in patients with FTD. The aim of this study is to determine how administration of intranasal oxytocin to patients with frontotemporal dementia affects behavior and processing of specific types of social and emotional information.The investigators' hypothesis is that oxytocin administration will improve emotional and social cognitive deficits in patients with FTD, resulting in improved decision making and behaviour.
Background: - Niemann-Pick disease type C (NPC) is a genetic disorder that results in progressive loss of nervous system function by affecting the membranes of nerve cells. There is no known cure for NPC. - N-acetyl cysteine (NAC) is a drug that has been approved by the Food and Drug Administration to use either orally or IV for the treatment of acetaminophen (Tylenol) poisoning or as an aerosol to reduce the stickiness of mucous in patients with cystic fibrosis. In the body, NAC is converted to an amino acid called cysteine, which cells can convert to a chemical called glutathione. Glutathione is important in helping cells deal with oxidative stress. Based on a number of experiments in cells, mice and patients with NPC, we believe that oxidative stress is increased in NPC. If we can increase glutathione levels, we may be able to decrease the oxidative stress. Objectives: - To test the safety and effectiveness of N-acetyl cysteine to treat Niemann-Pick disease (type C). Eligibility: - Individuals at least 1 year of age who have been diagnosed with NPC. Design: - Patients entering this study will be seen at the National Institutes of Health Clinical Center four times during the 20 weeks of the study. These admissions will occur at the start of the study and at weeks 8, 12, and 20. The first NIH visit will last 2 days, and the other visits will last 1 day. - Patients will participate in a two-stage study: a period of 8 weeks receiving NAC and a second period of 8 weeks when receiving a placebo (a pill without NAC). Every patient participating in this study will receive NAC during one of the two time periods. - The two treatment periods will be separated by a wash-out period, 4 weeks when patients will receive neither NAC nor placebo. Patients will also have a 4-week wash-out period at the beginning of the study. Most physician-prescribed medications, such as seizure medications, will be allowed. - During each visit, examinations, procedures, and tests will be done, including blood and urine samples.
Progressive aphasia is characterized by a steady and progressive loss of language skills in the presence of relatively preserved memory, attention, and thinking. The aim of this study is to slow the progression of language decline in progressive aphasia via language therapy. The first goal of this study is to improve naming abilities of individuals with progressive aphasia. This will be accomplished by carrying out an intensive treatment program for anomia. The second goal is to evaluate whether this intense language treatment re-activates affected areas and/or connections within the language network, using functional Magnetic Resonance Imaging (to measure neural activity in specific brain regions) and Diffusion Tensor Imaging tractography (to measure the connectivity between specific brain regions). This is the first study on progressive aphasia addressing both treatment and imaging in the same patients.
This is a prospective, open label, non-therapeutic, diagnostic imaging study. The purpose of this study is to utilize Pittsburgh Compound B positron emission imaging (PiB PET) to ascertain the relationship between change in amyloid burden over time, and concurrent change in clinical status.