Amyotrophic Lateral Sclerosis Clinical Trials
A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis Jacksonville, FL. This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral Sclerosis. After completion of Week 8, patients enrolled in a treatment group that is considered safe and …. Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year Jacksonville, FL. This study is to assess the effect of tirasemtiv versus placebo on respiratory function in patients with ALS. A Study to Assess The Amyotrophic Lateral Sclerosis Patient Experience Jacksonville, FL. The purpose of this study is to support and extend the results of the FORTITUDE-ALS clinical trial, a clinical trial of an investigational drug for the treatment of amyotrophic lateral sclerosis. A Study of the Safety and Effectiveness of NP001 for Patients with Amyotrophic Lateral Sclerosis and Systemic Inflammation Scottsdale/Phoenix, AZ. The purpose of this study is to assess the safety and effectiveness of NP001 for the treatment of patients who have amyotrophic lateral sclerosis and evidence of systemic inflammation. A Study of New Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis Rochester, MN. The purpose of this study is to evaluate and rank-order a set of new outcome measures administered to patients with amyotrophic lateral sclerosis, in order to identify measures that are more sensitive to disease progression than the current Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised. A Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis Scottsdale/Phoenix, AZ. This is a multicenter, multiple dose study to examine the effect of H.P. Acthar® on functional decline in adult subjects with amyotrophic lateral sclerosis. A Study of the Safety and Effectiveness of NP001 for Patients with Amyotrophic Lateral Sclerosis and Systemic Inflammation Jacksonville, FL. The purpose of this study is to evaluate the safety and effectiveness of NP001 for treating patients who have amyotrophic lateral sclerosis and evidence of systemic inflammation. A Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Participants With Amyotrophic Lateral Sclerosis Jacksonville, FL. This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis to explore safety, tolerability, and pharmacokinetic properties of GDC-0134. A Study to Evaluate the Effectiveness, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation Jacksonville, FL; Rochester, MN. The primary purpose of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics of BIIB067 in adults with Amyotrophic Lateral Sclerosis. A Study of the Safety of Mesenchymal Stem Cells for Treatment of One Patient with Amyotrophic Lateral Sclerosis Rochester, MN. The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of one patient who has amyotrophic lateral sclerosis. A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis Jacksonville, FL. The primary purpose of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis. Perampanel Transcranial Magnetic Stimulation in Amyotrophic Lateral Sclerosis Jacksonville, FL. The purpose of this study is to evaluate the potential to use TMS as a way of functionally assessing target engagement in an efficacy study of Perampanel as a treatment for ALS. The study will also determine if there is a correlation between Perampanel dose and TMS MT. Radicava® Biomarker Study in Participants with Amyotrophic Lateral Sclerosis Jacksonville, FL. The clinical development program has demonstrated that Edaravone is a well-tolerated treatment that slows the loss of physical function in ALS.However, as observed in prior clinical trials, ALS presentation and progression is extremely heterogeneous among patients. A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis Rochester, MN. The purpose of this study is to determine determine the safety of intraspinal delivery of mesenchymal stem cells to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis using a dose-escalation study.
Amyotrophic Lateral Sclerosis: External Research
Pre-disease biomarkers of persistent organic pollutants, immune system, and amyotrophic lateral sclerosisThe research study from the Harvard School of Public Health will measure the levels of persistent organic pollutants in blood samples of persons with ALS that were collected before the onset of ALS to learn if pre-ALS POPs levels are associated with the risk of developing ALS and survival with ALS. The POPs levels will be measured from pre-ALS blood samples taken from men and women who participated in Finnish and Danish health and diet surveys. The research study will then explore if changes to immune response cells in these samples occurred prior to ALS onset and, if so, learn whether these changes to the immune response cells resulted from POPs exposures or caused ALS. The study hypothesizes that higher pre-ALS POPs levels increase the risk of developing ALS, decrease survival with ALS, and cause changes to immune response cells in persons with ALS. 2. Serological profiling of the human virome and ALS risk in a military populationThe research study from the Harvard T.H. Chan School of Public Health will examine blood samples collected from healthy U.S. armed forces service members, including persons who later developed ALS and persons who remained healthy. A novel innate immunity risk factor for amyotrophic lateral sclerosisThe research study from the Northwestern University at Chicago will look for genetic variants of an innate immunity protein called apolipoprotein L1 in DNA and RNA samples of North American ALS patients. The research study will explore whether these ALS patients had past trypanosome infections and then evaluate the relationship between ALS variant expression, past trypanosome infections, and environmental factors, including occupational and geographic exposure to trypanosomes. Environmental risk factors for ALS: critical time periods and genetic interactionsThe research study from Dartmouth College will investigate the most susceptible time periods during which environmental exposures carry the greatest risk for later development of ALS, by using residential history to estimate exposures to waterbody cyanobacteria, pesticides, and polluted sites in ALS patients compared to healthy controls in time-linked databases. The research team will also develop a pooled genotype dataset derived from genetic variants implicated in neurodegenerative illness on ATSDR National ALS Biorepository and Dartmouth College northern New England control and ALS specimens to evaluate gene x environment interactions in ALS risk. Metabolomic Signatures Linking ALS to Persistent Organic Pollutant ExposuresThe research study from the University of Michigan proposes to identify the metabolomic signatures that correlate with persistent organic pollutant exposures and historical exposure risk factors, and determine whether these are associated with ALS progression. The study will be the first to evaluate whether central nervous system derived extracellular vesicles can be potentially used as novel biomarkers of environmental exposures and disease progression in ALS. The research investigators will test biospecimens from persons with ALS for metals and non-persistent pesticides, while matching exposure- and patient-specific transcriptomic signatures to ALS signaling pathways. Identification and Characterization of Potential Environmental Risk Factors for ALS Using the ATSDR ALS Registry Cases and a Control PopulationThe research study will examine environmental toxicants from ambient air pollution in persons with ALS from the ATSDR National ALS Registry and non-ALS controls using information in blood and provided from self-report questionnaires on occupational and non-occupational exposures. We will also look at the effect of that relationship on how likely a person is to develop ALS. This study will be done thanks to the Clinical Research in ALS and related disorders for Therapeutic Development Consortium that is a part of the Rare Diseases Clinical Research Network. Large-Scale Genome-Wide Association Study of ALS. The goal of this project is to identify genes that might be responsible for amyotrophic lateral sclerosis.