ALS Research Notification for Clinical Trials and Studies – Closed
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1. A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants with ALS
The purpose of the Engensis ALS Study is to evaluate the safety and tolerability of Engensis in 18 ALS patients. The trial will assess muscle function and strength using the ALS Functional Rating (ALSFRS-R) and Hand-Held Dynamometry (HHD). Muscle biopsies will also be collected to identify biomarkers for future research. Engensis will compared to a placebo. Participation will last approximately 6 months and will require 11 visits to a physician’s office. All study participants will be randomly assigned in a 2:1 ratio to Engenis or placebo.
2. Survey of communication methods, interventions, and participation for people with ALS
The purpose of this study is to learn more about the communication methods used by people with ALS, how people with ALS learn about their options for communication, and how people with ALS participate in various communication situations. The information gathered for this study will be used to help improve the communication options and communication support services available to people with ALS. We want to hear from all people with ALS, regardless of whether they have experienced speech changes, or what methods they use for communication. This one-time survey will take approximately 30-60 minutes. Participants may complete the survey online, or may request a paper survey. Responses will be kept anonymous. We aim to recruit at least 376 people for this study.
To learn more about this study click here, email firstname.lastname@example.org or call 503-494-2732
3. ALS Patient’s Resilience, Self-Determination, and Decision-Making for Life-Sustaining Treatments
The purpose of this study is to increase knowledge related to the health care decision-making of people with ALS (pALS). By doing this study, the investigator hopes to better understanding of how PALS experiences with, and their attitudes about resilience are related to their decision-making for life-sustaining treatments, such as tracheostomies and feeding tubes. The results from this study will inform how ALS care teams communicate with their patients about life-sustaining treatments and could justify funding and resources for people who choose to get tracheostomies and feeding tubes. The recruitment goal is 250 participants and the survey will take approximately 30 minutes to complete.
4. A Study of the Symptoms of Greatest Importance in Motor Neuron Disease (MND)
The purpose of this research is to determine the issues and symptoms that are most important to patients with ALS. The information from this study will ultimately be used to help develop an ALS-specific quality of life instrument to be used in future clinical trials and patient care settings. Anyone with a diagnosis of ALS is able to participate. We are looking for approximately 7000 patients to participate.
5. Phase 3 Safety and Efficacy of NurOwn in ALS
The purpose of this study is to test if repeated dosing of NurOwn® (mesenchymal stem cells/MSC) a type of cell derived from your own bone marrow (the stem cells-containing fluid inside your bones), is safe without causing too many side effects in people with ALS and whether it may decrease the rate of decline of the disease. MSCs are specialized cells which are made to release chemicals to support the nervous system. To do this, study doctors will remove some of your bone marrow cells, treat them in a laboratory to become MSCs, and put them back into the fluid that surrounds your spinal cord. It will take up to 12 months to complete the research study. During this time, we will ask you to make 14 study visits to the study site. During the study, you will be required to spend three nights as an inpatient for a 24 hours follow-up visit after each administration of NurOwn®.
6. Safety of Urate Elevation in ALS – Phase II (SURE-ALS2)
We are doing this research study to find out if inosine is safe to take without causing too many side effects in people with ALS. Inosine is as an over-the-counter supplement. Taking inosine causes an increase in the blood levels of urate (uric acid). Urate is a natural antioxidant present in the body that may help cancel out oxidative stress. The FDA has not approved inosine to treat ALS. It will take you about 27 weeks to complete this research study. This is a double-blind, placebo controlled study. We will test your breathing, collect blood and ask you questions about your past medical history and medications you are taking. You will be required to have and use a smartphone to participate in this study as this study will utilize a mobile application to collect information on tasks that you perform while using your smartphone.
7. RDCRN Contact Registry for the CReATe Consortium
Patients with ALS and related diseases enroll in research at a low rate. These patients may be unaware of research opportunities. The CReATe Contact Registry is a way for patients with ALS and related disorders to learn about CReATe research studies. These studies will gather basic information about the patient. The purpose of this research study is to gather information about a patient and his/her child’s disease. This registry helps connect patients with researchers. It is not a population-based registry that quantifies the disease.
8. A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in ALS
This is a Phase 3, Randomised, placebo controlled trial on arimoclomol in ALS. The aim of this trial is to find out if arimoclomol extends the ability of independent breathing, improves survival, functional health and safety. The trial is open to adult patients (18 years and older) that are diagnosed with ALS by a neurologist and experienced first symptoms of weakness (e.g. weakness in limbs, difficulty swallowing, difficulty speaking or shortness of breath within the last 18 months. An ALSFRS-R of 35 and SVC of 70% of predicted normal is required. The trial is 18 months in duration requiring regular clinic visits (every 8-12 weeks) where your health and function is monitored. It is required that blood, urine and CSF are collected in this trial.
9. The ALS Association Care Services Survey
This survey supports our care services program. Our care services program helps patients and families cope with ALS by providing information, resources, and referrals to services. Every question in the survey helps us (ALS Association) the services we deliver or could deliver to people with ALS. It will also help us understand challenges people may face in accessing care or living with the disease.
10. Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, CENTAUR-ALS
This research study is to find out whether the drug AMX0035 works safely to slow down the worsening of symptoms in people with ALS, including muscle strength. 132 people with ALS will be enrolled to take part in the study. Taking part means participating in up to seven in-person study visits and four phone call visits over about seven months. People with ALS who participate will take study drug for about six months. During the study visits, the researchers will take blood samples, perform basic medical check-up activities, ask participants to complete questionnaires and undergo procedures to measure the participants’ breathing and muscle strength. These procedures to measure breathing and muscle strength and activity should not result in any pain or major discomfort. Learn more:https://www.nejm.org/doi/full/10.1056/NEJMoa1916945
11. Effects of Oral Levosimedan (ODM-109) on Respiratory Function in Patients with ALS
The REFALS study will confirm that levosimendan can significantly improve respiratory function measured by supine slow vital capacity (SVC) in amyotrophic lateral sclerosis (ALS) patients. Approximately 450 participants will be participating in approximately 70-80 study centers in countries throughout the world. Participants will be in the study 51-52 weeks and visit the study clinic 10 times. Both males and females, over 18 years of age are eligible to participate. Patient must be able to swallow pills and be diagnosed with ALS.
12. A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With ALS (Fortitude-ALS)
This is a multi-national, double-blind, randomized, placebo-controlled, parallel group study in patients with ALS with the selective fast skeletal muscle troponin activator, CK-CK-2127107. Enrolled participants will be dosed with CK-2127107 150, 300, 450 mg or placebo twice daily (300, 600, 900 mg/day or placebo) for a period of 12 weeks.
13. Skill‐based dysphagia therapy as an intervention for individuals with Neurodegenerative disease.
Through this research, we will gain more information about the impact of swallowing skill training on ability to eat and quality of life for people with ALS. There are currently no well researched swallowing therapies for people with ALS. We will collect information about swallowing and participant reports of quality of life before and after the treatment sessions. This research will include 24 participants with ALS. Participants must be above 18 years of age and diagnosed with ALS with no other conditions that may affect their swallowing. Participants must be able to follow directions to complete the therapy. Participants must have noticed some changes to their swallowing because of ALS but still be able to eat a variety of foods.
14. 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.
To learn more about this study click here
15. 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.
16. 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.
17. Mexiletine in Sporadic Amyotrophic Lateral Sclerosis
This research study is to find out whether the drug mexiletine works to calm over-excited nerves and brain cells in people with ALS. We hope that this will also slow down how quickly the disease advances. 60 people with ALS will be enrolled to take part in the study. Taking part means participating in up to four study visits and one telephone call over about eleven weeks. You will receive study drug for about four weeks. During the study visits, the researchers will take blood samples, perform basic medical check-up activities, ask you to complete questionnaires, and undergo procedures to measure your breathing, and muscle and brain activity. These procedures measuring your breathing and muscle and brain activity should not result in any pain or major discomfort.
18. A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps
Muscle cramps are involuntary, painful muscle contractions with sudden onset lasting seconds to several minutes. While muscle cramps usually resolve spontaneously or with stretching, many patients with underlying neurological or metabolic disease suffer from frequent or sustained cramping which can lead to persistent muscle soreness and swelling after cramp occurrence. This study will assess the efficacy of FLX-787-ODT versus Low Dose Control over a 28-day treatment period for treatment of muscle cramps in subjects with MND. Approximately one hundred and fifty (150) male or female subjects will be screened to achieve approximately 90 subjects to complete the study.
19. A Phase 2 Randomized, Placebo-Controlled Trial of Tocilizumab in Amyotrophic Lateral Sclerosis (ALS)
The purpose of the research study is to determine whether tocilizumab (ActemraTM) is safe and tolerable for patients with Amyotrophic Lateral Sclerosis (ALS). This study will be conducted in subjects who meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS. At screening, eligible subjects must be at least 18 years old, must have a slow vital capacity (SVC) ≥ 40% of predicted capacity for age, height and gender. Approximately 24 subjects will be randomized in the study across 5 NEALS centers in the US. While taking the study drug, participants will be asked to complete several tests and questionnaires. From this study, the researchers hope to learn more about ALS and its treatment. Study visits will be completed over 16 weeks. Participants must be at least 18 years of age. Participants should be located close to the study site and be able to follow study tasks.
20. IMPACT-ALS (Investigating and Measuring Patient And Caregiver Trends about ALS)
The survey is intended to identify the burden of ALS and concerns during the course of the disease, preferences for treatment, and any differences in perceptions between patients and caregivers. Results from this survey are planned to be published in peer review journals and other channels. Results will be accessible to all participants.
21. Treatment of FUS-Related ALS with Betamethasone – The TRANSLATE Study
The ALS Center at the University of Kentucky (Lexington KY) is conducting a study for ALS patients with genetic mutations in the FUS gene. Certain genetics factors can cause these incorrect mutations. A drug called betamethasone may benefit patients with these mutations. This study will look at the drug interactions of patients who have this particular FUS mutation while taking betamethasone and test if the drug restores antioxidant activity.
22. 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.
23. 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.
24. 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
25. 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.
26. 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.
27. 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.
28. 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.
29. 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.
30. Identification and Validation of ALS Environmental Risk Factors.
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.
31. 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.
32. 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.
33. 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.
34. 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.
35. 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.
36. 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.
37. 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.
38 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.
39. 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
40. 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.
41. 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.
42. 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.
43. 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.