ALS Projects Supported via the Registry’s Research Notification Mechanism

  • ALS Testing through Home-Based Outcome Measures
  • It is important to track disease progression in ALS to improve research and treatment. Disease tracking can be used to evaluate how a patient is doing. These evaluations may require participants to visit a clinic. This travel requirement may prevent participation in studies due to travel distance or mobility issues. The ALS AT HOME study aims to address the problem of travel. Participants will be taught to take their own measurements at home. We hope that ALS patients will evaluate their own function at home. This self-evaluation should increase participation in trials. We also hope that increasing the frequency of measurements will improve test consistency.

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  • Microbiome Assessment in People with ALS
  • ALS is a complex disease that is not well understood. There is growing interest in the role of gut microbiota in the development of diseases such as ALS. These organisms in the gut may contribute to inflammation in ALS. This study will investigate the role of microbiota in ALS. The study will compare gut composition in people with ALS to healthy people through genetic tests. The study will also examine the role of gut composition in ALS progression.

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  • A Phase 2 Study of NP001 in Subjects with ALS and Evidence of Elevated Systemic Inflammation
  • Neuraltus is conducting a phase 2 study of NP001 in ALS patients. The study will determine if NP001 slows down ALS symptoms in patients with increased inflammation in their blood. Patients will receive NP001 or a placebo. They will receive these treatments for 5 days in a row during the first month and for 3 days in a row in months 2 through 6. In month 7 there will be an end-of-study visit. The effects of NP001 will be explored by functional measures, time to tracheotomy, and inflammatory biomarkers. More information can be found at: https://clinicaltrials.gov/ct2/show/NCT02794857?term=np001&rank=3

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  • Biospecimen Collection to Investigate the Causes of ALS.
  • A study is being conducted on ALS patients and ALS patients with frontotemporal dementia (FTD) that contain a risk factor in a gene named c9ORF72. These two groups with this risk factor are called c9ALS and c9ALS/FTD. This study seeks to understand c9ORF72’s role in ALS. The study will take place every 6 months for up to 5 years. Participants will be evaluated on performance of daily activities, cognitive and behavior function, and muscle strength. Blood samples will also be collected to monitor the disease and evaluate response to treatment.

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  • A Prospective Comprehensive Epidemiologic Study in a Large Cohort in The National ALS Registry: A Step to Identify ALS Risk Factors.
  • The goal of this study is to examine the relationship between oxidative stress (OS) and ALS. OS is shown by a combination of risk factors along with increased levels of urine OS biomarkers, and ALS disease progression, shown by function and survival. This study investigates the effect of combined exposures on development of ALS, including environmental, occupational, lifestyle, dietary, and psychological risk factors. All of these factors may increase systemic OS in patients with ALS. The hypothesis is that patients who have greater OS have faster disease progression. The relationship between OS and ALS has never been investigated.

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  • A Spatial Analysis of ALS in Florida, Ohio, New Hampshire, and Vermont.
  • The goal of this study is to use spatial analysis to evaluate the distribution of ALS cases in FL, OH, NH, and VT. Authors will look for areas of higher than expected disease incidence. They will also examine the spatial correlations with environmental toxicants such as heavy metals and cyanobacterial toxins. Using the questionnaire, each person taking part will be asked for a list of all current and past residential addresses, including place of birth. The populations of OH, FL, NH, and VT with ALS will be the main focus of this study. The general population that does not have a neurodegenerative disease will be used as the control group.

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  • The Natural History and Biomarkers of C9ORF72 ALS and Frontotemporal Dementia (FTD).
  • The main goal of this study is to describe the natural history of ALS in patients who carry a repeat expansion in the C9ORF72 gene. This will be done using standard clinical assessments. A second goal is to assess possible biomarkers of disease progression. The authors will evaluate motor, cognitive, and behavioral functions every 6 months over 3 years. This will include 4 in-person visits to NIH in Bethesda, Maryland, and phone assessments. The study population consists of 62 adults, 18 years of age or older, who have the repeat expansion of the C9ORF72 gene confirmed by certified testing. The study includes three main outcome measures: the ALS Functional rating scale, the fronto-behavioral inventory, and a verbal fluency index. The authors will also look at forced vital capacity and survival. Exploratory biomarkers include imaging, physiology, spinal fluid, blood, and skin biopsies. The authors will study the link between primary and secondary clinical outcome measures and candidate biomarkers. This will be done in an exploratory way to see if candidate biomarkers are predictive of disease onset or progression.

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  • VITALITY-ALS (Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices after Treatment for a Year)
  • Cytokinetics is conducting a Phase 3 clinical trial for the drug Tirasemtiv. Tirasemtiv is a drug that affects skeletal muscle receptors. It is for ALS patients that have muscle weakness and fatigue. Tirasemtiv selectively activates the fast skeletal muscle receptors and increases their sensitivity to calcium. This results in increased skeletal muscle force and power. It also delays the time to muscle fatigue. More information about the clinical trial can be found at: https://www.clinicaltrials.gov/ct2/show/study/NCT02496767?term=Vitality+ALS&rank=1&show_locs=Y#locn.

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  • Methodology Study of Novel Outcome Measures to Assess Progression of ALS.
  • This study measures the progression of ALS among persons 18 to 80 years of age who have been diagnosed in the past two years. Patients who participate will be asked to complete questionnaires and assessments in order to determine ALS progression. The study lasts for 24 months. Patients will be asked to come in for study visits every three months for the first year. They will also have additional monthly telephone calls for the first six months. In the second year of the study, patients will only have telephone calls every three months. The information gathered from this study will help provide more accurate and consistent outcome measures for future ALS research. This could ultimately help new ALS drugs get to market.

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  • Identification and Validation of ALS Environmental Risk Factors. University of Michigan.
  • This study has two goals. The first goal is to identify potential environmental risk factors associated with ALS. These risk factors could be exposures to toxicants in the environment or at work as well as physical exertion. Subjects will include ALS patients in the University of Michigan’s ALS Clinic and Michigan residents in the National ALS Registry. It will also include a control group. The control group will be persons without ALS who are matched on age and sex. Results from a detailed questionnaire including work and home exposures will be analyzed. The researchers will also use estimates of exposure to geographical pollutants and measure exposure biomarkers in biospecimens. These datasets will be analyzed to identify potential risk factors associated with ALS. The authors hypothesize that ALS risk may be elevated among individuals exposed to toxicants such as pesticides and/or selected fertilizers. Also, it is possible that physical exertion is a risk factor or effect modifier. The second goal is to use biomarkers to evaluate exposures using information from on questionnaires and environmental assessments. Comparisons will be made between ALS patients and control groups. Potential exposures will be assessed using the survey instrument. These exposure estimates will also use geographic information systems, environmental assessments, and biomarker data. Studying how risk factors vary with critical exposure time periods has significant potential to improve our understanding of disease pathogenesis. It could also help identify new biomarkers to aid in ALS diagnosis.

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  • A Phase 2 Pharmacodynamic Study of Ezogabine on Neuronal Excitability in Amyotrophic Lateral Sclerosis.
  • This study is being done to understand more about motor neurons in people who have ALS compared with people without ALS. This is multi-center, randomized, double-blind, placebo- controlled clinical trial. The study lasts 14 weeks. We will evaluate the effect of ezogabine treatment on neuronal excitability in ALS subjects. Clinic visits will occur at Screening, Baseline, and Weeks 4, 6, 8, and 12. There will also be two phone call visits in between clinic visits and one last phone call visit at week 14. Persons who are 18 years of age or older, with Slow vital capacity ≥ 50% of expected, can take part.

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  • A Study to Evaluate the Sensitivity, Specificity, and Overall Accuracy of an Amyotrophic Lateral Sclerosis Diagnostic Test.
  • The diagnosis of ALS currently relies on clinical evaluation. This can cause a delay in diagnosis of up to 18 months. We have identified specific biomarkers in CSF and blood. These biomarkers are related to the disease state of ALS for diagnosis and prognosis. The focus of this study is to see if these biomarkers can tell the difference between ALS and other neurological diseases. The primary objective of the study is to see how a CSF-based test works for ALS diagnosis. We will recruit a group from a defined geography area in the US and Europe. The collected biosamples will also be used to enable research for new biomarkers in the future. To take part, you must have been recently diagnosed with sporadic or familial ALS and other neuromuscular diseases. The diagnosis must have occurred within 18 months of symptoms on-set. You must also be between 21 and 80 years of age.

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  • Evaluating the safety tolerability and clinical endpoint responsiveness of Ibudilast MN 166 in subjects with ALS.
  • This is a single center, randomized, double-blind, placebo-controlled study of Ibudilast. The study will take 6 months, followed by open-label treatment. The first objective is to see if Ibudilast is safe and what side-effects it might have compared with taking a placebo. The study drug will be taken for six months in addition to Riluzole. The second objective is to see whether there are changes in functional activity, respiratory function, muscle strength, and use of non-invasive ventilation. The third objective is to look at quality of life, a biomarker for disease progression, and how long Ibudilast stays in the body.

    About 60 patients will be enrolled. Subjects must be 18 to 80 years old. They can have sporadic or familial ALS. Disease onset must have occurred in the last 3 years before screening. Patients must be on a stable dose of Riluzole for at least 1 month before study drug treatment starts. They must have a slow vital capacity ≥ 60% of predicted within 1 month before the first day of treatment. Patients must have gotten pneumococcal vaccine within the last four years.

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  • Amyotrophic Lateral Sclerosis and Genetic Testing: A Perspective from the ALS Community
  • The purpose of this study is to ask patients with ALS about their understanding and experience with genetic testing. Any ALS patient in the ATSDR's National ALS Registry may take part. Information will be collected on patient access to and experience with genetic testing. The study will focus on why patients seek genetic testing and whether or not it is helpful. Results from this study may help ALS clinicians better serve the needs of ALS patients. This project may be a starting point for the development of guidelines for genetic counseling and testing in ALS.

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  • Speech Motor Impairments
  • This study will collect data from persons with ALS and other motor neuron diseases. Eighty-four participants with ALS and fifty-one healthy controls will be recruited. All of those who take part will be 35 to 80 years old. Each ALS participant will be asked to make up to 5 visits to the lab over about 2.5 years. Those who take part will be asked to do several things such as make a variety of facial expressions for an evaluation, drink several sips of water, and chew gum. They will also be asked to answer several health form and quality of life surveys. The results of this study expect to improve early detection and accuracy of ALS diagnosis. It will help find objective outcome measures for ongoing drug trials. It will also provide data to develop a novel oral communication device for persons with moderate to severe speech impairment. This device will be able to recognize impaired movement and translate that movement into functional speech.

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  • ALS Quest: An online questionnaire for research into ALS
  • This is an international study looking for factors in the environment that may lead to ALS. The survey is aimed at finding which risk factors for ALS are present in individual countries. It will also look at which factors are common to many countries. People in different countries will be able to read and answer the questions in their own languages. The study is centered at the University of Sydney in Australia. People who take part in the survey will not be identified because no personal information is given. People both with and without ALS are being asked to take part. Anybody aged 18 years or older can complete the survey.

    The survey can be taken using any type of computer at www.alsquest.org. A guide starts with how to complete the survey, followed by a wide range of questions about possible risk factors for ALS. The survey can be completed over a number of sessions, and can be run with software used to assist people with physical disabilities to enter data. We hope the information that arises from the survey will find risk factors that will help find measures to prevent and treat ALS.

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  • Multi-Center, Randomized Controlled Study of the NeuRx® Diaphragm Pacing System™ (DPS) In Participants with Amyotrophic Lateral Sclerosis (ALS)
  • Diaphragm weakness is a significant contributor to respiratory problems and respiratory failure in people with ALS. This study looks at whether treatment with the NeuRx® Diaphragm Pacing System™ (DPS) helps people with ALS and hypoventilation.

    The primary goal of this study is to compare standard of care to DPS with respect to survival. The secondary goal is to compare measures of diaphragm function, dyspnea, and quality of life between the standard of care group and DPS group.

    This randomized, controlled clinical trial will compare standard of care treatment to DPS in people with ALS and hypoventilation. There will be 7 clinic visits. These clinic visits will occur at screening and on Months 3, 6, 9, 12, 15, and 18. Study participants will receive monthly phone calls between clinic visits for the duration of the study.

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  • Assessing Pain in Amyotrophic Lateral Sclerosis
  • This is a cross-sectional observational study that uses an anonymous on-line survey. The goal of this study is to examine how depression, anxiety and self-efficacy influence pain severity, pain impact on daily life, and pain relief.

    We will use the National ALS Registry to recruit ALS patients who report any kind of pain. We will conduct this study using a study website. Those who take part will be informed about the procedures, benefits, and risks of the study. We will collect data on demographics. We will also ask participants to complete the ALS Functional Rating Scale-R, Brief Pain Inventory, Hospital Anxiety and Depression Scale, and Chronic Pain Self-Efficacy Scale.

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  • Questionnaire of cramps and pain in ALS
  • Muscle cramps affect the majority of people with ALS. This study wants to estimate the proportion of ALS patients troubled by cramps related to how long they have had ALS. It also wants to estimate the severity and impact of cramps in ALS patients. Study participants will be asked to complete an online questionnaire. This questionnaire will include a series of standard questions about pain and cramps. Data will be analyzed using traditional methods. Researchers will also calculate the correlation between symptoms and disease onset.

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  • VA Biorepository Brain Bank ALS Study
  • Studies show that ALS is more common among veterans than civilians. The Department of Veterans Affairs (VA) started the VA Biorepository Brain Bank (VABBB) to support ALS research. The VABBB is a human tissue bank. It collects, processes, stores and gives out nervous system tissue specimens for future studies. The VABBB is enrolling Veterans in the United States who suffer from ALS. It is also enrolling Veterans with diseases related to ALS. Those diseases include primary lateral sclerosis (PLS), progressive bulbar palsy (PBP), and progressive muscular atrophy (PMA). Veterans are enrolled if they would like to make an after-death brain and spinal cord donation. In addition, Veterans enrolled in the VABBB are asked to complete telephone and/or mail surveys about their health every six months or so. The VABBB makes all arrangements for the tissue donation. The donation process occurs at no cost to the Veteran’s family.

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  • Developing a Satellite ALS Center at a Remote Site Incorporating Regional Resources and Telemedicine.
  • The goal of this pilot study is to develop and evaluate a model of care delivery for ALS patients who reside in remote, underserved areas of the country. This will be called a "Virtual ALS Center" (VALSC). The authors will see if multidisciplinary ALS care can be given at a remote, community-based site in a way that can be kept up and is cost-efficient. The authors will develop a VALSC in the Appalachian region, centered in Ashland Kentucky. The VALSC will give care in a multidisciplinary ALS clinic setting for ALS patients from the region. This will be done in ongoing fashion using videoconferencing between the VALSC and the ALS Certified Center at the University of Kentucky. The authors will compare the care given to a group of pre-VALSC ALS patients residing in the target region with a matched group of ALS patients receiving their care at the ALS Certified Center at the University of Kentucky. The authors will measure the success of the VALSC in giving care at their regional site. The authors will also evaluate the total costs and barriers to delivering multidisciplinary care in a VALSC. If the study is successful, it could serve as a generalizable model to deliver ALS care in remote areas of the country. It could also establish relationships with affiliated VALSCs to collaborate on future clinical drug trials in ALS.

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  • The Experimental Treatment of Bulbar Dysfunction in ALS.
  • The goal of this study is to see whether Nuedextra helps speech, swallowing and salivation in persons with ALS. The authors will measure whether there is a significant change in self-report bulbar function scale (CNS-BFS) score. This is a double-blind, randomized, controlled, cross-over study. Each person who takes part will take either Nuedextra or placebo for 30 days, and then take Nuedextra for additional 30 days. There are 5 in-person visits. A number of questionnaires/tests will be done at each visit. This study is being done in subjects with a clinical diagnosis of ALS. The persons must meet the El Escorial criteria for possible, laboratory-supported probable, probable, or definite ALS. The primary efficacy variable is the CNS-BFS score and will be measured at baseline and throughout the study.

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  • Mexiletine for the treatment of muscle cramps in ALS.
  • This study looks at whether mexiletine is effective in reducing the number of muscle cramps in ALS. This will be assessed in 30 subjects in a double-blinded six-week crossover study. Cramp intensity will also be examined. The authors will also evaluate mexiletine's effect on muscle fasciculations. Fasciculations are tracked along with muscle cramps using similar methods. The authors will also evaluate side effects from mexiletine in ALS patients with cramps. The authors think the mexiletine will help cramps and fasciculations. The authors also do not think that there will be significant side effects that make a person with ALS stop the drug. This study is conducted at UC Davis, and its more than 100 affiliated telemedicine physician offices throughout the state of California as well as UC San Diego, UCLA and UC Irvine

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  • Mindfulness, Psychological Well-Being, and Physical Degeneration in People with ALS.
  • This study investigated the relationship among mindfulness, quality of life, anxiety, depression and changes in physical impairment in persons with ALS. A sample of 197 subjects with ALS took part. Each person who took part was assessed online twice; an initial assessment and four months later. The authors measured traits of mindfulness, physical impairment, quality of life, anxiety, and depression. Mindfulness positively influenced physical symptoms. Subjects with higher mindfulness scores had slower progression of the disease after four months. Mindfulness at first assessment predicted higher quality of life and psychological well-being at the second assessment.

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  • Phase II/III, Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1+ Familial ALS.
  • The goal of this trial was to test the safety and efficacy of a novel compound: arimoclomol. The authors tested the compound in patients with rapidly progressive familial ALS due to mutations in the SOD1 gene. Phase II assessed the safety and tolerability of arimoclomol over a 6-month period. Phase III examined whether chronic treatment with arimoclomol slowed disease progression over a 12-month period as measured by the ALSFRS-R. Safety and tolerability were evaluated. The rate of decline of ALSFRS-R over 12 months; expiratory volume in 6 seconds (FEV6); and time to death, tracheostomy or permanent assisted ventilation were measured. The analysis included comparison of (1) rate of decline of ALSFRS-R and FEV6 in the two treatment groups and (2) tracheostomy-free survival in the two treatment groups.

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  • Risk Factor Analysis in ALS.
  • This study will look at risk factors for ALS. Persons with ALS and persons without ALS (controls) will fill in web based surveys. The authors will look at environmental and occupational exposure to chemicals. The chemicals include metals (i.e., lead and mercury), pesticides, and solvents. The authors will also look at personal risk factors such as excessive physical activity, history of head trauma, and family history of neurologic disorders. Lastly, the authors will look at exposure to neurotoxicant ambient air pollutants estimated by where you have lived. Additionally, the study will investigate race/ethnic differences in relation to risk of ALS.

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