ACTIVE – ALS Research Notification for Clinical Trials and Studies

The following are approved ALS studies and clinical trials that have used the National ALS Registry for patient recruitment purposes. Email notifications have already been sent out to PALS meeting the specific study criteria.

Active: currently recruiting for the specified clinical trial or study
To view list of closed (recruiting has ended) clinical trials and studies click here .

To receive automatic notifications about the latest clinical trials and studies, please join the Registry, click here.

This list will be updated as new research proposals are approved by ATSDR.

ALS Research Notification Clinical Trials Studies
No Study Name Institution Investigator Research Notification Date* Information Link Status
1 Rasch-built Outcome Measures to Improve ALS Clinical Trials Emory University Christina Fournier, MD
New!
APR 2022
Click herepdf icon Active
2 Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS Mitsubishi Tanabe Pharma Development America, Inc. Multiple
New!
APR 2022
Click hereexternal icon Active
3 Phase 2a Study of TPN-101 in Patients with C9ORF72 ALS/FTD (Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia) Transposon Therapeutics Merit Cudkowicz, MD
New!
MAR 2022
Click hereexternal icon Active
4 Home-based Remote Digital Monitoring to Assess ALS progression Emory University Jonathan Glass, MD
New!
FEB 2022
Click hereexternal icon Active
5 COURAGE-ALS Phase 3 Clinical Trial Cytokinetics, Inc Stacy A. Rudnicki, MD
New!
FEB 2022
Click hereexternal icon Active
6 Radicava®/(Edaravone) Findings in Biomarkers From ALS Mitsubishi Tanabe Pharma James Berry, MD
New!
FEB 2022
Click hereexternal icon Active
7 Microbiome in the Progression of ALS Emory University Vicki Hertzberg, PhD
New!
JAN 2022
Click hereexternal icon Active
8 Evaluation of IONIS in FUS-ALS Patients Ionis Pharmaceuticals Multiple
New!
JAN 2022
Click hereexternal icon Active
9 COVID-19 ALS Registry Atrium Health Urvi Desai, MD
DEC 2021
Click hereexternal icon Active
10 ALS Focus Survey Program ALS Association Sarah Parvanta, PhD
DEC 2021
Click hereexternal icon Active
11 Identify, Analyze, and Evaluate Potential Risk Factors for Amyotrophic Lateral Sclerosis (ALS) Dartmouth-Hitchcock Medical Center Elijah Stommel, MD, PhD
OCT 2021
Email
Ph:866-894-8131
Active
12 The E-health Application to Modify Oral Energy intake and Measure Outcomes Remotely in ALS Massachusetts General Hospital Anne-Marie Wills, MD
MAY 2021
Click hereexternal icon Active
13 ALS Quest: An online questionnaire for research into ALS University of Sydney Roger Pamphlett, MD, MB
APR 2021
Click hereexternal icon Active
14 Answer ALS Companion App Johns Hopkins University SOM Jeffrey Rothstein, MD, PhD
MAR 2021
Click hereexternal icon Active
15 Feasibility of the BrainGate2 Neural Interface System in Persons with Tetraplegia Massachusetts General Hospital Leigh R. Hochberg, M.D. Ph.D
FEB 2021
Email Active
16 Measuring self-reported fatigue in people with MND and healthy volunteers. Massachusetts General Hospital James Berry, MD
NOV 2020
Click hereexternal icon Active
17 A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients Alexion Pharmaceuticals Glen Frick, MD, PhD
NOV 2020
Click hereexternal icon Active
18 VA Biorepository Brain Bank ALS Study VA Boston Healthcare System Neil W. Kowall, MD
OCT 2020
Click hereexternal icon Active
19 Experiences of symptomatic and at-risk individuals with genetic testing for ALS (Amyotrophic Lateral Sclerosis) Emory University School of Medicine Lauren Lichten, MS
AUG 2020
Click hereexternal icon Active
20 Impact of COVID-19 on People Living with Rare Disease and their Families University of Cincinnati Maurizio Macaluso, MD, DrPH
JUL 2020
Click hereexternal icon Active
21 Determining the half-life of SOD1 in the CSF Washington University (St. Louis) Timothy Miller, MD, PhD
DEC 2019
Email
Ph:844-ALS-CARE
Active
22 Psychosocial Impact of Genetic Testing in ALS University of Chicago Raymond Roos, MD
NOV 2019
Click hereexternal icon Active
23 Infant Feeding Characteristics of Patients with Amyotrophic Lateral Sclerosis (ALS) University of North Carolina at Chapel Hill Karsey Erickson
JAN 2019
Click hereexternal icon Active
24 ALS Quest: An online questionnaire for research into ALS University of Sydney Roger Pamphlett, MD, MB
NOV 2018
Click hereexternal icon Active

* Date most recent email was sent.

ALS Projects Supported via the Registry’s Research Notification Mechanism

1. Rasch-built Outcome Measures to Improve ALS Clinical Trials
Many ALS research studies use questionnaires or scales to track disease progression. The purpose of this study is to validate and create new scales for measuring ALS symptoms. Adults aged 18 or older with a diagnosis of ALS are eligible to participate. Study participants will complete questionnaires about their ALS every month for a minimum of 6 months. Participants will also complete questionnaires 1 time by phone and will have 2 neurologic exams by video teleconference. No in-person visits are required. This study is not expected to benefit participants directly, but we hope this research will allow us to conduct more efficient and decisive ALS research studies in the future.
To learn more about this study, email click here pdf icon[PDF – 268 KB]

2. Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS
This research study is being conducted to better understand an investigational medication called edaravone in an oral/by mouth formula. Approximately 380 participants (190 to each group) will be enrolled around the world. Study participation is approximately 58 weeks. This includes a screening period (up to 8 weeks), a treatment period for 48 weeks, and a safety follow-up period of 2 weeks. Participants must have been diagnosed with Amyotrophic Lateral Sclerosis (ALS), had their first symptom of ALS within 2 years, are able to take medications orally (by mouth), and have not received any previous treatment of edaravone. This study is a randomized and double-blind research study. “Randomized” means you will be randomly assigned by chance (like flipping a coin) to one of the 2 treatment groups of the study. “Double-blind” means neither you nor the study doctor/staff will know to which treatment group you are assigned. In the event of an emergency, the study doctor can quickly find out your treatment group.
To learn more about this study, email click hereexternal icon

3 Phase 2a Study of TPN-101 in Patients with C9ORF72 ALS/FTD (Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia)
Nerve cells in patients with ALS due to a mutation in the C9orf72 gene produce harmful DNA, RNA, and proteins. These products cause the cells to become inflamed and damaged. These effects can be blocked by inhibiting an enzyme in the cell called LINE-1 reverse transcriptase. TPN-101 is a new inhibitor of this enzyme that is being tested in this study. We hope to learn whether TPN-101 can reduce the damage to nerve cells and improve the symptoms of patients with ALS. 40 patients with a mutation in the C9orf72 gene will participate in this study. They will be randomly assigned to receive either TPN-101 (60% of the participants) or an inactive placebo (40% of the participants). TPN-101 is an oral capsule taken once a day. We will compare the group that gets TPN-101 to the group that gets placebo to see if the patients on TPN-101 do better on symptom scales and on tests done on blood and cerebrospinal fluid. After the first 6 months of treatment, the patients taking placebo will be able to take TPN-101 for 6 months. The total duration of each patient’s participation in the study is one year. During this time, patients will come to the clinic 12 or 13 times. Each visit will last between 2 hours and half a day. Patients will have clinical assessments, blood tests, and 3 lumbar punctures to obtain the cerebrospinal fluid from a needle inserted into the lower back.
To learn more about this study, email click hereexternal icon

4. Home-based Remote Digital Monitoring to Assess ALS progression
The ALS disease course can only be modestly modified by current therapies and clinical trials are necessary to find more and better therapies to treat the disease. There is a burden associated with frequent travel to the hospital, hindering the ability of many ALS patients to participate in trials. The purpose of this study is to see whether we can acquire at-home measurements that are dependable and can help with tracking disease progression.
If you participate in this study, you do not have to come to the clinic and all assessments can be accomplished at home. We hope the information we gain from this study will help improve future clinical trials and help change the way ALS patients are cared for.
To learn more about this study, email click hereexternal icon

5. COURAGE-ALS Phase 3 clinical trial
COURAGE-ALS is a phase 3 placebo-controlled clinical trial researching the safety and efficacy of an investigational drug called reldesemtiv. The main goal of the clinical trial is to learn if taking reldesemtiv will slow the decline in the progression of ALS.
The clinical trial participation will be over a 54-week period. It will include a screening, treatment (48 weeks), and a follow-up period for all participants. The first 24 weeks of the treatment period will compare the disease progression for participants taking reldesemtiv compared to placebo (2:1) All participants completing the first 24 weeks and entering second 24 weeks of the treatment period will receive reldesemtiv (active drug only, no placebo).
To learn more about this study, email click hereexternal icon

6. Radicava®/(Edaravone) Findings in Biomarkers From ALS
Radicava has been shown to slow the loss of physical function in ALS and was approved by the FDA in May of 2017 as a treatment for ALS. In this research study we want to learn what changes happen in patients with Amyotrophic Lateral Sclerosis (ALS) that can be seen in the blood and urine when taking Radicava. These measurable indicators of change are called biomarkers. This study hopes to learn more about the biology of ALS, disease progression, and the treatment effect of Radicava by studying biomarkers. The study plans to enroll up to 300 patients who will take Radicava over a 24 week period. Participants are to complete 6 cycles of Radicava infusion and provide blood and urine for analysis. You may be eligible for this study if you have been diagnosed with ALS, are 18 years or older and after discussion with your physician, the decision was made to prescribe Radicava. Study participants will obtain drug through their own current medical/pharmacy coverage plan (i.e. Medicare, HMO, PPO, etc.).
To learn more about this study, email click hereexternal icon

7. Microbiome in the Progression of ALS
Emerging research indicates that the gut microbiome (the bacteria, fungi, yeasts and viruses that live in our gut) may have a role in ALS, specifically a role in disease progression. We propose to identify genera / species of microbes that are deficient in ALS patients and/or those that are overabundant and possibly deleterious. In this way we can, in a later study, determine if replenishment of these microbes could slow disease progression and/or if the deleterious microbes represent therapeutic (antibiotic) targets. In this project we will evaluate changes in gut and oral microbiota over time in ALS patients and their partner / caregiver controls. We are recruiting 30 Patients and their partner/caregivers to participate in this study. Participants much agree to participate as a couple. Patients must be no more than 6 months from diagnosis and 18 months from symptom onset and must not be on a feeding tube at baseline. All participants must have no medical history of bowel disease (except for only constipation) (i.e., Crohn’s disease, irritable bowel disease); no medical history of malignancy; no medical history of dementia, Parkinson’s disease, Alzheimer’s disease, other neurodegenerative or cognitive disorder; and no instance of gastrointestinal infection and/or diarrhea in the last week.
To learn more about this study, email click hereexternal icon

8. Evaluation of IONIS in FUS-ALS Patients
The primary purpose of this study is to evaluate the clinical efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS). IONIS hopes to obtain more information on the efficacy, safety, pharmacokinetics and pharmacodynamics of the study ION363 in patients suffering from the rare FUS genetic mutation associated with ALS.
To learn more about this study click hereexternal icon

9. COVID-19 ALS Registry
Measuring self-reported fatigue in people with MND and healthy volunteers Fatigue is a prevalent symptom in patients with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases.
To learn more about this study click hereexternal icon

10. ALS Focus Survey Program
ALS Focus is patient- and caregiver-led survey program from The ALS Association that asks people with ALS, as well as current and past caregivers, about their needs and preferences throughout their disease journey. We are proud that every step of the survey development process is informed and reviewed by a committee of people with ALS and caregivers.

The goal is to learn as much as possible about individual experiences, needs, and challenges, so that the entire community can benefit. All information collected is de-identified and shared free of charge to researchers from all over the world and other organizations working on ALS. We are forming robust and accurate data showing the experiences and needs of people with ALS and their families. Open data helps us strengthen care for people with ALS and caregivers, accelerate therapy development, improve clinical trials, influence policy makers, and more.
For more information on ALS Focus and participating in surveys, please visit www.als.org/als-focus/external icon.

11. Identify, Analyze, and Evaluate Potential Risk Factors for Amyotrophic Lateral Sclerosis (ALS)
This study aims to identify, analyze, and evaluate environmental risk factors for ALS. ALS patients residing predominantly in Ohio and New England are encouraged to participate. The study is also open to residents in the continental UnitedStates. Participants will complete an environmental exposure history questionnaire and provide blood and/or saliva. Reimbursements will be provided upon receipt of a completed questionnaire or biological samples.
To learn more about this study, email neuroresearch@hitchcock.org or call 866-894-8131

12. The E-health Application to Modify Oral Energy intake and Measure Outcomes Remotely in ALS
The goal of this study is to design, test and disseminate a nutrition app specially designed for people with ALS. The majority of people with ALS around the country do not have access to dietitians. Therefore we wish to create an app which can be used widely by people with ALS. We aim to evaluate an ALS mobile health app that we designed, which enables nutritional counseling and monitoring of weight and disease progression.
To learn more about this study, email mghALSapp@partners.org or call 617-643-2400

13. 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.orgexternal icon. 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 hereexternal icon

14. Answer ALS Companion App
Due to the nature of ALS, PLS, and other related motor neuron diseases, it often becomes more difficult for patients to travel to sites to participate in research studies. The use of smartphone applications (apps) in clinical practice and clinical trials has opened up possibilities for tracking disease progression without frequent visits to a center. This app requires a different task to be performed once per day for an indefinite amount of time. You will use it for as long as you are able or until you no longer want to participate. Tasks include voice, breathing, and fine motor exercises. You will also be asked complete the ALS Functional Rating Scale-Revised (ALSFRS-R) at periodic intervals.
To learn more about this study click hereexternal icon

15. Feasibility of the BrainGate2 Neural Interface System in Persons with Tetraplegia
The goal of the BrainGate2 Clinical Trial is to see if a new medical device called the BrainGate2 Neural Interface System (also called “BrainGate”) is safe and effective at giving people with paralysis the ability to control a computer cursor and other assistive devices with their thoughts.
To learn more about this study, email clinicaltrials@braingate.org

16. Measuring self-reported fatigue in people with MND and healthy volunteers
Fatigue is a prevalent symptom in patients with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases. There is an urgent need to understand and reliably measure fatigue in people with motor neuron diseases. This will help to determine whether therapies are able to treat this symptom and benefit patients. The purpose of this study is to measure patient reported fatigue in people with motor neuron diseases, to compare reported fatigue in people with motor neuron diseases with fatigue in healthy volunteers, and to track changes in fatigue over time as it relates to function.
To learn more about this study click hereexternal icon

17. A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients
The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS. This is a is a randomized, double-blind, placebo-controlled trial. Participants will be randomly assigned to receive ravulizumab or placebo. The study has a randomized controlled period of 50 weeks and during this time, the participant and the research team do not know who is receiving placebo or ravulizumab. After the randomized controlled period, participants can enter the Open-label Extension Period of the study for up to 2 years and receive ravulizumab.
To learn more about this study click hereexternal icon

18. 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.
To learn more about this study click hereexternal icon

19. Experiences of symptomatic and at-risk individuals with genetic testing for ALS (Amyotrophic Lateral Sclerosis)
Most cases of Amyotrophic Lateral Sclerosis (ALS) are sporadic, but approximately 5-10% of ALS can be hereditary or inherited. Genetic testing can determine if an affected individual has hereditary ALS. If so, this individual’s family members can consider having genetic testing to determine if they also will develop ALS. The objective is to evaluate the experiences of individuals who have ALS and at-risk individuals undergoing genetic testing for ALS along with assessing whether published guidelines are followed for at-risk blood relatives when offered genetic testing for ALS; to improve clinical genetics services in ALS. The participants of the study include individuals affected with ALS and at-risk individuals who were offered genetic testing for ALS.
To learn more about this study  click hereexternal icon

20. Impact of COVID-19 on People Living with Rare Disease and their Families
The novel coronavirus pandemic is impacting people with rare diseases and their families. We want to find out how many people who have a rare disease have become sick with COVID-19, how COVID-19 has affected someone who also has a rare disease, their symptoms, their treatments and their access to care. We also want to learn how the COVID-19 pandemic is affecting the lives of people with rare diseases who have NOT been sick with COVID-19. The pandemic may have caused stress, anxiety,and made access to care harder, especially in areas where COVID-19 is more common. We want to figure out how the Rare Diseases Clinical Research Network (RDCRN) can respond to those concerns by perhaps providing information or advice through its consortia and patient advocacy groups. It should take you approximately 20 minutes to complete.
To learn more about this study click hereexternal icon

21. Determining the half-life of SOD1 in the CSF
Washington University in St. Louis is seeking participants with familial or sporadic ALS for a study to determine the half-life of the protein SOD1 in the cerebral spinal fluid. Mutations in the SOD1 gene are known to cause some forms of familial ALS. Researchers are developing a treatment to reduce the level of SOD1 in familial ALS, but need to know more about how long SOD1 stays in the body (“half-life”) to help determine if the new treatment is effective. The study involves labeling or marking SOD1 with a special type of leucine. Leucine is an essential amino acid that is found in the foods we eat. In order to label the protein, participants undergo either an overnight IV infusion or eat a controlled diet for 10 days. Following this labeling process, participants will then have 4-5 lumbar punctures (spinal taps) over a period of 3-4 months to collect spinal fluid from which the half-life of SOD1 can be measured.
To learn more about this study, email ALS@wustl.edu or call 844-ALS-CARE

22. Psychosocial Impact of Genetic Testing in ALS
The University of Chicago is currently recruiting patients who have a diagnosis of ALS to participate in a research study that involves a one-time survey. The purpose of this study is to better understand how people with ALS make decisions with regard to obtaining or forgoing genetic testing as well as the impact of such testing. This study will also ask questions about your attitudes and thoughts on advance care planning and end-of-life decisions. This study will help clinicians better understand and address the needs of patients and families with ALS.
To learn more about this study click hereexternal icon

23. Infant Feeding Characteristics of Patients with Amyotrophic Lateral Sclerosis (ALS)
The purpose of this survey is to look at the prevalence of different infant feeding methods among people with ALS. There is currently no available data to support an association of infant feeding practices and the development of ALS. This is the first survey to investigate such an avenue.
To learn more about this study click hereexternal icon

24. 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.orgexternal icon. 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 hereexternal icon

Page last reviewed: April 26, 2022