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 Designing a Resource of Accessible, Creative Technologies University of Washington Erin Beneteau
New!
JUL 2021
Click hereexternal icon Active
2 Evaluation of IONIS in FUS-ALS Patients Ionis Pharmaceuticals Neil Shneider, MD, PhD
New!
JUL 2021
Click hereexternal icon Active
3 ALS Focus Survey Program ALS Association Sarah Parvanta, PhD
New!
JUN 2021
Click hereexternal icon Active
4 Radicava®/(Edaravone) Findings in Biomarkers From ALS Mitsubishi Tanabe Pharma James Berry, MD
New!
JUN 2021
Click hereexternal icon Active
5 Microbiome in the Progression of ALS Emory University Vicki Hertzberg, PhD
New!
MAY 2021
Click hereexternal icon Active
6 The E-health Application to Modify Oral Energy intake and Measure Outcomes Remotely in ALS Massachusetts General Hospital Anne-Marie Wills, MD
New!
MAY 2021
Click hereexternal icon Active
7 ALS Quest: An online questionnaire for research into ALS University of Sydney Roger Pamphlett, MD, MB
APR 2021
Click hereexternal icon Active
8 Answer ALS Companion App Johns Hopkins University SOM Jeffrey Rothstein, MD, PhD
MAR 2021
Click hereexternal icon Active
9 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
10 COVID-19 ALS Registry Atrium Health Urvi Desai, MD
JAN 2021
Click hereexternal icon Active
11 A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants with ALS Helixmith Co., Ltd Yessar M. Hussain, MD
JAN 2021
Benjamin Joslin,
Study Coordinator
Email
Ph:312-503-7504
Active
12 Measuring self-reported fatigue in people with MND and healthy volunteers. Massachusetts General Hospital James Berry, MD
NOV 2020
Click hereexternal icon Active
13 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
14 VA Biorepository Brain Bank ALS Study VA Boston Healthcare System Neil W. Kowall, MD
OCT 2020
Click hereexternal icon Active
15 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
16 Identify, Analyze, and Evaluate Potential Risk Factors for Amyotrophic Lateral Sclerosis (ALS) Dartmouth-Hitchcock Medical Center Elijah Stommel, MD, PhD
JUL 2020
Email
Ph:866-894-8131
Active
17 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
18 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
19 Psychosocial Impact of Genetic Testing in ALS University of Chicago Raymond Roos, MD
NOV 2019
Click hereexternal icon Active
20 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
21 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. Designing a Resource of Accessible, Creative Technologies
This survey is focused on accessibility for people who have motor, mobility and/or communication disabilities and are interested in creativity. We want to learn from four different groups of people who are interested in accessible creativity:
1) Adults who have motor, mobility, and/or communication disabilities and who use Assistive Technologies.
2) Caregivers/Carepartners of people who have motor, mobility, and/or communication disabilities and who use Assistive Technologies.
3) Therapists and Clinicians who work with people who have motor, mobility, and/or communication disabilities and who use Assistive Technologies.
4) Technology Designers and Developers who build creative technologies and/or build Assistive Technologies
To learn more about this study click hereexternal icon

2. 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

3. ALS Focus Survey Program
ALS Focus is a patient- and caregiver-led survey program that asks people with ALS as well as current and past caregivers about their needs and burdens. 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 so that the entire community can benefit. All information collected is de-identified and shared free of charge, which provides The ALS Association, researchers from all over the world, and other organizations working on ALS with accurate information around the disease journey. This will help us strengthen care for people with ALS and caregivers, accelerate therapy development, improve clinical trials, influence policy makers, and more.
ALS Focus has opened a new Caregiver Needs survey. We invite current and former caregivers to visit www.alsfocus.orgexternal icon today to share their experiences. Responses to this survey will help the ALS community find ways to improve support for caregivers. The Caregiver Needs survey is for current and former caregivers and will take just five minutes to complete after registering. If you are not a caregiver but you have one in your life, please let them know about this survey! For more information on ALS Focus, visit www.als.org/als-focus/external icon.
To learn more about this study click hereexternal icon

4. 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

5. 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

6. 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

7. 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

8. 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

9. 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

10. 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

11. 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.
To learn more about this study,  email Benjamin Joslin  or call 312-503-7504

12. 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

13. 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

14. 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

15. 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

16. 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

17. 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

18. 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

19. 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

20. 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

21. 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 14, 2021