Active clinical trials for "Motor Neuron Disease"

The study is being conducted to determine if DPS treatment for people with ALS and hypoventilation is associated with improved survival or diaphragm function. The primary objective of the study is to conduct a multi-center, randomized controlled clinical trial comparing standard of care (control) to diaphragm stimulator treatment with the NeuRx® Diaphragm Pacing System™ (DPS) with respect to survival. The secondary objective of the study is to conduct a multi-center, randomized controlled clinical trial to compare standard of care treatment (control) to DPS in ALS subjects with hypoventilation.

The purpose of this research project is to collect and store blood samples and clinical data. Researchers can then use the stored samples in future studies. Through such studies, they hope to find new ways to detect, treat, and maybe even prevent or cure health problems.

The purpose of this study is to collect 650 blood and 300 cerebrospinal fluid (CSF) samples from people with amyotrophic lateral sclerosis (ALS), pure lower or upper motor neuron diseases, as well as other neurodegenerative diseases and from people with no neurological disorder. Through comparison of these samples, the researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to diagnose ALS and monitor disease progression. Additionally, up to 600 blood samples will be collected for a sub-study for DNA analysis. Studying components of the blood, such as DNA, may help us understand what happens when genes function abnormally and how it might be related to disease.

Amyotrophic lateral sclerosis is a uniformly progressive and fatal neurodegenerative disorder for which there is no known cure. In a novel attempt to widen the search for potential therapeutic agents, a NINDS- led cooperative group performed an in-vitro screening program of 1040 FDA approved drugs in over 28 assays relevant to various neurodegenerative disorders. Several cephalosporins showed hits in ALS relevant assays. Efficacy was noted in models suggesting increased expression of the astrocytic glutamate transporter, EAAT2, as well as models of superoxide dismutase mediated toxicity. Ceftriaxone is a third generation cephalosporin with good CNS penetration, a long half-life, and was effective in both types of ALS assays. Ceftriaxone has calcium binding activity, antioxidant properties, and rescues motor neurons in culture from chronic glutamate toxicity. Since completion of the original NINDS screen, Ceftriaxone has been shown to increase by three fold EAAT2 activity in rodent brains, due to ceftriaxone's ability to increase EAAT2 promotor activation This program is for the use of ceftriaxone in ALS for compassionate care. Currently ceftriaxone is approved by the U.S. Food and Drug Administration (FDA) for treating bacterial infections but not for treating ALS. However, there is an ongoing phase I study -by NEALS Consortium and the National Institute of Health- with three cohorts -a placebo group and two groups receiving either 2 or 4 grams of ceftriaxone daily-. Unfortunately there are only a limited number of patients being enrolled and the next phase of the project will not be undertaken until next year. At this point there are ALS patients unable to participate in this Phase I trial and unlikely to be alive when the next phase of study begins. Some of these patients want to receive the drug and are willing to pay for the drug and nursing care. We are therefore requesting a compassionate use protocol for these patients who request the medication and are willing to pay for the drug and nursing care to administer it. Dr. Terry Heiman-Patterson will supervise the administration and safety monitoring including labs for renal and hepatic function as well as IV site inspection.

Results about effects of symptomatic treatment on QoL appears conflicting in ALS patients. Moreover no clear effects of gastrotomy have be shown on survival. Prospective study on effect of tube feeding, QoL and survival is performed in 17 teaching hospitals in France (observational study)

This study is a follow-up to an earlier study that examined the relationship of This study will examine whether exposure to neurotoxins, such as lead, mercury, solvents, and pesticides, can contribute to amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. The cause of this degenerative disease of the brain and spinal cord is not well understood. Some studies suggest that exposure to environmental neurotoxins may increase its risk. This follow-up study will examine the relationship of neurotoxin exposure to the interval between the diagnosis of ALS and death. It will also examine the possible roles of genetics, lifestyle and dietary factors in the disease. Information on ALS patients previously enrolled in the study will be used to examine this relationship. No new individuals will be enrolled in the study.

This is a single-center, phase 2 randomized clinical trial of tamoxifen on mean percent predicted isometric muscle strength in patients with amyotrophic lateral sclerosis (ALS). The purpose is to determine whether the triphenylethylenetamoxifen, used as adjuvant therapy in the treatment of breast cancer, can delay the loss of isometric muscle strength in ALS patients.

The purpose of this study is to assess the functional mobility and self-reported satisfaction with the Xavier electromyography hands-free wheelchair control system in comparison with a standard joystick.

Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e. a "reversal." Thirty-eight of these patients were identified in the prior Duke University study, Documentation of Known ALS Reversals (St.A.R. Protocol 1, Duke IRB Pro00076395). The investigators hypothesize these patients have had different environmental exposures than patients with typically progressive ALS. Identification of specific environmental influences may point to exposures which are protective or exposure that lead to the development of a rare and novel reversible ALS-like disease. Objective: This study seeks to identify environmental exposures associated with ALS reversals.

The specific objective of this study is to validate the practice of remote pulmonary function testing (rPFT) conducted in the home through the use of connected mobile health devices and the Penn State Hershey ALS Telemanagement program.