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.
Background: - Frontotemporal dementia (FTD) is the second most common cause of early-onset dementia. Alzheimer s disease is the most common. Alzheimer s disease happens most often in the elderly, but FTD typically appears between 40 and 60 years of age. It also has a strong genetic component: Up to 40% of FTD cases are linked to positive family histories. Earlier diagnoses and genetic tests mean that people with FTD will spend more years in earlier stages of disease, aware that they have it. However, few studies have looked at the personal experiences or coping styles of people with FTD. Researchers want to interview people with FTD and their caregivers to understand their experiences with the disease. This information will help create better treatments and therapies for those affected by FTD. Objectives: - To study the experiences of persons with FTD and their primary caregivers. Eligibility: - Individuals at least 18 years of age who have been diagnosed with FTD. - Primary caregivers (spouse or partner at least 18 years of age) of individuals who have been diagnosed with FTD. Design: - Before FTD participants are recruited, a pilot study will test the interview questions. This pilot study will be given to people with Alzheimer s disease and their caregivers. It will study how well people with dementia understand the interview questions. - FTD study participants will be recruited through dementia care centers. - All participants will have in-person interviews. These interviews will take up to 1 hour. - Participants with FTD will answer questions about their experience with the disease. They will talk about their mental abilities, challenges, and coping strategies. - Caregivers will answer questions about their experience in caring for someone with FTD. They will talk about their challenges and coping strategies. They will also talk about the person with FTD, and how aware they believe that the person is of the dementia symptoms. - All participants will receive a small gift card as compensation for their time. - No treatment will be provided as part of this study.
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 study is to use a functional MRI (fMRI) index to compare the brain activity of healthy volunteers to that of people with mild cognitive impairment (MCI) and Alzheimer's disease. The ultimate goal is to develop an early diagnostic tool for Alzheimer's disease. The study hypotheses are: 1. The fMRI index will differentiate between Alzheimer's disease, non-Alzheimer's dementia, and healthy volunteers; 2. The fMRI index will distinguish participants with MCI who convert to Alzheimer's disease from those who convert to a non-Alzheimer's dementia and those who remain stable; 3. MCI participants with a lower fMRI index at baseline who convert will progress to Alzheimer's sooner than those with a higher fMRI index, and MCI participants with a faster rate of fMRI index decline who convert will have an earlier onset of Alzheimer's disease.
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.
The investigators laboratory has been studying families with a history of ALS for more than 30 years and is continuing to use new ways to understand how genes may play a role in ALS, motor neuron disease and other neuromuscular disorders. The purpose of this study is to identify additional genes that may cause or put a person at risk for either familial ALS (meaning 2 or more people in a family who have had ALS), sporadic ALS, or other forms of motor neuron disease in the hopes of improving diagnosis and treatment. As new genes are found that may be linked to ALS in families or individuals, the investigators can then further study how that gene may be contributing to the disease by studying it down to the protein and molecular level. This includes all forms of ALS, motor neuron disease and ALS with fronto-temporal dementia(ALS/FTD). We also continue to study other forms of neuromuscular disease such as Miyoshi myopathy, FSH dystrophy and other forms of muscular dystrophy by looking at the genes that may be associated with them. There have been a number of genes identified that are associated with both familial and sporadic ALS, with the SOD1, C9orf72, and FUS genes explaining the majority of the cases. However, for about 25% of families with FALS, the gene(s) are still unknown. The investigators also will continue to work with families already identified to carry one of the known genes associated with ALS.
Tau pathology and tangles have been associated with cognitive dysfunction causing neurodegeneration. AD, the most abundant tauopathy is characterized by amyloid plaques and tau tangles. An abundance of tau inclusions, in the absence of amyloid deposits, defines Pick's disease (frontotemporal lobar degeneration), progressive supranuclear palsy (PSP), corticobasal degeneration (CBD) and other diseases including frontal atrophy associated with cognitive clinical dysfunction of frontal dysexecutive syndrome, progressive nonfluent aphasia and semantic dementia as recently reviewed (Gozes 2010). It is the investigators aim to follow other protein expression [as per recent publications (Marksteiner et al., 2011)] in blood and CSF samples from those tauopathies. Significance: Results should establish the possibility of using tau and other proteins as markers for early detection and disease progression in FTD, also in comparison to Alzheimer's disease (AD).