View clinical trials related to Pick Disease of the Brain.
Filter by:Background: - Hydroxypropyl beta cyclodextrin (HPBCD) is being tested for a disease called Niemann-Pick disease type C1 (NPC1). NPC1 is a genetic disorder that results in gradual loss of nervous system function. Cholesterol and other fats have trouble moving out of the brain cells, which makes the cells work poorly and leads to symptoms. There is no treatment currently approved in the US for NPC1. Researchers want to test if it is safe to use HPBCD for NPC1. They want to see if it can help brain cells process cholesterol better. Objectives: - To test the safety and effectiveness of HPBCD for NPC1. Eligibility: - Individuals between 2 and 25 years of age who have been diagnosed with NPC1 and who have not already received HPBCD in an attempt to treat NPC1. Design: - Participants will be screened with a physical exam and medical history. They will provide blood and urine samples for screening. They will also have neurological tests, including tests of hearing, speech and movement. - Participants will have a lumbar puncture (also called a spinal tap) every month to deliver the drug to the spinal fluid that surrounds the brain. The length of the trial will be determined by the safety and efficacy information that is obtained. - Treatment will be monitored with frequent blood and urine tests, cerebral spinal fluid tests, hearing and neurological exams.
The study is designed to assess the demographic, clinical and imaging associations with the presence of microbleeds in atypical Alzheimer's disease. The primary hypothesis is that cognitive and functional performance will be poorer in atypical Alzheimer's subjects with microbleeds compared to those without microbleeds.
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 study is designed to determine whether there are clinical features that can be used as biomarkers to predict whether underlying Alzheimer's pathology is the cause of a speech and language based dementia. The primary hypothesis is that the proportion of patients who test positive for beta-amyloid deposition will vary across different speech and language based dementias.
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.
The purpose of this study is to assess the performance of AclarusDx™, an investigational blood test detecting gene expression information, and intended to help physicians in making an Alzheimer's Disease diagnosis in patients having memory impairments.
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 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.