View clinical trials related to Progressive Supranuclear Palsy.
Filter by:The main goal of the GENEPARK consortium is to employ innovative haemogenomic approaches to determine gene expression profiles specific for genetic and idiopathic Parkinson's disease (PD) patients. These gene expression signatures will be utilised clinically as non-invasive diagnostic tests for PD. The sensitivity of the newly developed diagnostic test will be determined by extensive validations on an independent cohort of PD patients, whereas the specificity will be assessed by testing patients with atypical parkinsonisms, including multiple system atrophy, progressive supranuclear palsy and diffuse Lewy body disease. In order to test the specificity of the diagnostic set in other disorders that affect basal ganglia, Huntington's disease and dopa responsive dystonia patients will be analysed. The second objective of the proposal is to determine correlations between gene expression signatures and different stages of PD and thus provide the basis for early diagnosis and monitoring of disease progression. These changes in blood gene expression will be correlated with alterations detected by neuroimaging in the brain of PD patients. Such combinations of molecular and morphological markers of disease may ultimately facilitate the selection and monitoring of neuroprotective therapies for PD. Finally, GENEPARK aims to develop new bioinformatic software tools for selection of genomic biomarkers using microarray data. A set of established computational tools will be applied and novel methods, some of them based on mechanistic modelling of the neurodegenerative diseases, will be developed in order to study the advantages and limitations of the different methodologies. With special emphasis on the careful clinical selection of patients and sufficient power regarding patient numbers, as well as extensive quality control and validation of the data, GENEPARK aims to develop a standardised approach to development and validation of haemogenomic biomarkers of disease.
Progressive supranuclear palsy (PSP) is the most common atypical parkinsonian movement disorder. This study will determine the role of specific genetic, occupational and environmental components in the development of PSP by evaluating patients with this disorder and age and gender matched controls.
Progressive Supranuclear Palsy (PSP) is a relentlessly progressive neurodegenerative disorder, clinically characterized by parkinsonism with prominent axial involvement and postural instability, bulbar symptoms, supranuclear ophthalmoplegia, and executive dysfunction. Abnormal neuronal and glial tau aggregations affecting the basal ganglia and selective brainstem structures result in dysfunction of the five frontosubcortical circuits and brainstem functions. There is no effective treatment for PSP. One of the key feature in the aggregation of tau is its phosphorylation by kinases such as glycogen synthase kinase 3b (GSK3b). Recent reports have shown that valproic acid was able to inhibit the activity of GSK3b and could exert a neuroprotective effect through this inhibition. The investigators thus decided to conduct this controlled study to assess the putative neuroprotective effects in patients with PSP.
The clinical syndrome of PSP responds poorly to all available forms of therapy used in Parkinson's Disease (PD). Currently, no effective treatment exists. Coenzyme Q10 in high doses has been shown to be a beneficial therapy in PD and might possibly be a beneficial therapy for PSP. This study will compare the efficacy, safety and tolerability of Coenzyme Q10 versus placebo in patients with atypical parkinsonian syndrome, progressive supranuclear palsy (PSP).
Study hypothesis: A 6-week p.o treatment with 5 mg/Kg Coenzyme Q10 is safe and tolerable,increases the brain's metabolism and ameliorates clinical symptoms in patients with PSP.
This study will examine the safety and effectiveness of an experimental drug called GDNF delivered through an investigational device to treat progressive supranuclear palsy (PSP). The drug will be administered directly into the brain through catheters attached to an infusion pump implanted in the abdomen. The study will evaluate 1) if the drug is safe and well tolerated when given by this method; 2) the performance of the catheters and pump system, and 3) the effects of GDNF on PSP symptoms. PSP is a rare neurological disease that causes eye movement dysfunction, muscle rigidity, slowness of movement, swallowing, speech, emotional, cognitive and personality problems. Patients 35 to 75 years old with PSP may be eligible for this study. Candidates will be screened at the National Institutes of Health outpatient clinic in Bethesda, MD, with a medical history, physical examination, neurological and neuropsychiatric evaluations, blood tests, electrocardiogram, CT scan of the brain, and baseline studies including a special eye examination, evaluation of symptoms, lumbar puncture (spinal tap) and psychiatric interview. Patients enrolled in the study will undergo surgery to place two catheters into the brain and two infusion pumps under the skin in the upper abdomen. The surgery will be performed at Vanderbilt University Medical Center in Nashville, TN. It will be done under general anesthesia and will require a 3 day hospitalization. Within 24 hours after the surgery, a CT scan of the brain will be done to ensure the catheters are properly placed. Patients return to NIH two weeks after surgery for post-surgery examination and treatment initiation. All patients will receive continuous infusions of GNDF through one catheter and placebo (an inactive salt solution) through the other for 6 months. Half of the patients will receive placebo in the right side of the brain and GNDF in the left, and half will receive GNDF in the right side of the brain and placebo in the left. All patients will also undergo the following procedures: Brief physical examination, and evaluation of symptoms and adverse side effects - every 2 weeks Blood and urine tests - every 2 weeks for the first 2 months and then every 8 weeks until the end of the study CT scan to check catheter placement - weeks 9 and 27 Thorough evaluation of symptoms - before beginning treatment and weeks 1, 5, 9, 17 and 27 Neuropsychiatric evaluation - week 27 Special eye examination - weeks 1 and 27 Lumbar puncture - week 27 Additional blood tests to measure drug concentration and antibodies - 6 times during the study In addition, some patients may be asked to have positron emission tomography (PET) scans or a single photon emission tomography (SPECT) scan, or both. The potential benefit of GDNF is unknown. In studies with rats and monkeys, GNDF increased the number and size of brain cells containing the chemical messenger dopamine and some movement and balance problems were lessened. Earlier studies of GDNF infused into the ventricles of patients with Parkinson's disease showed no benefit and no serious harm.