If you're not sure which of these trials might be right for you, contact our ALS Research Access Nurse for guidance.

Enrolling ALS Clinical Trials

HEALEY ALS Platform Trial

Current Platform Regimens

Each regimen is being conducted to see if the specified investigational drug is a safe and effective treatment option for people diagnosed with ALS. 

Learn more about the platform trial.

Regimen A: Trial of Zilucoplan
This medication works by inhibiting tissue damage caused by the immune system.
Developed by: UCB

Regimen B: Trial of Verdiperstat
This medication works by reducing neural inflammation.
Developed by: Biohaven Pharmaceuticals

Regimen C: Trial of CNM-Au8
This medication provides an energetic assist to impaired motor neurons and helps improve their ability to function normally.
Developed by: Clene Nanomedicine

Regimen D: Trial of Pridopidine
This medication exerts neuroprotective effects via activation of the Sigma-1 receptor (S1R).
Developed by: Prilenia Therapeutics

Learn more about the study drugs

For participation at Massachusetts General Hospital:
Email: MGHsiteHealeyPlatform@mgh.harvard.edu
Phone: 617-643-3902

For participation at other sites, contact the Patient Navigator:
Email: HealeyALSplatform@mgh.harvard.edu
Phone: 833-425-8257

Trial of AT-1501

Full Trial Name: A Phase 2a Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Multiple Doses of AT-1501 in Adults with ALS
Trial Phase: 2
Principal Investigator: Sabrina Paganoni, MD, PhD
Sponsor: Anelixis Therapeutics, Inc.

We are doing this research study to find out about the safety and tolerability of the study drug AT-1501. The study is funded by Anelixis Therapeutics Inc. This study is recruiting patients diagnosed for 24 months or less with Amyotrophic Lateral Sclerosis (ALS) with an ALSFRS-R score of 35 or higher. Participation in this study will last for 19 weeks and will include up to 7 visits to Massachusetts General Hospital. There are additional inclusion/exclusion criteria that the study team will review with you in more detail if you are interested in participating. For more information regarding this trial, or to further discuss how to participate, please contact the following study coordinators:
Zoe Scheier: 617-643-4803, zscheier@mgh.harvard.edu

Trial of BIIB05 for ALS and polyQ-ALS

Full Trial Name: A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 Administered Intrathecally to Adults with Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the Ataxin-2 Gene
Trial Phase: 1
Principal Investigator: . Suma Babu, MBBS, MPH
Sponsor: Biogen MA, Inc.

The purpose of this Phase 1 research study is to learn about the safety and tolerability of the study drug BIIB105 in adults with a diagnosis of Amyotrophic Lateral Sclerosis who have a slow vital capacity of ≥60%. This study will also look at the level and action of the study drug in your body, and what happens to this level over time. BIIB105 is administered intrathecally (via lumbar puncture into the lower spine). This study is placebo-controlled, which means that some participants will receive placebo, which looks like the study drug but does not contain any active ingredients.

Participation in the study will last for approximately 29 weeks, including a 4-week screening period, 13-week treatment period and a 12-week follow-up period. During the treatment period, participants will receive 3 loading doses of BIIB105 every 2 weeks, followed by 2 doses administered once every 4 weeks, for a total of 5 doses. For more information regarding this trial, please contact one of the study coordinators:
Mackenzie Keegan: 617-643-6252 or mkeegan@mgh.harvard.edu
Gabriel Jacobs, 617-726-3015 or gjacobs@mgh.harvard.edu

Trial of BLZ945 for ALS

Full Trial Name: -label, adaptive design study in patients with ALS to characterize safety, tolerability and brain microglia response, as measured by TSPO binding, following multiple doses of BLZ945 using positron emission tomography (PET) with radioligand [11C]-PBR28
Trial Phase: 2

This Phase II research study is being done to study the safety and tolerability of a molecule called BLZ945 in patients with ALS. We also want to find out if BLZ945 is safe to take without causing too many side effects in ALS. Novartis is the sponsor of this study, and BLZ945 is taken orally. This research study will use an imaging method known as Positron Emission Tomography or PET to measure the effect of BLZ945 on a specific inflammatory cell type in the brain called microglia, which are activated in ALS. The study aims at providing important information on whether BLZ945 could be a potential treatment for patients with ALS and to help to select the most appropriate doses for the planning of future research in patients with ALS. This study is open label, which means that all participants receive study drug. Study participation will last, at most, 84 days and include up to 5-7 hospital admission days at MGH. Please contact the study team to obtain additional information about the study visits and procedures. The study team will review with the inclusion and exclusion criteria during the study, which is also available on clinicaltrials.gov.

Principal Investigator: Suma Babu, MD, MPH
Sponsor: Novartis Enrollment
Enrollment Contacts:
Austin Lewis, 617-724-7928 or alewis29@mgh.harvard.edu
Mackenzie Keegan, 617-643-6252 or mkeegan@mgh.harvard.edu
Trial of RAPA-501 Cell Therapy for ALS

Full Trial Name: Phase I Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) for Amyotrophic Lateral Sclerosis
Trial Phase: 1

We are doing this Phase I, three-cohort research study to find out if RAPA-501 cell therapy is safe in patients with ALS. This is an open label study, which means that there is no placebo, and all participants will receive actual RAPA-501 cell infusions. Two doses of RAPA-501 cells will be investigated for safety. In addition, if RAPA-501 cells are found to be safe, additional patients will receive RAPA-501 cells in combination with a low dose of two chemotherapy agents. The study is looking for patients with a diagnosis of Amyotrophic Lateral Sclerosis who have a forced vital capacity of ≥ 50%. In this study, participants’ white blood cells (T-cells) will be removed during a procedure called Apheresis (a type of blood filtering). The T cells will be made into the specialized RAPA-501 cells and re-infused through an I.V. Total study participation will last about 1 year; 6 months of treatment (up to 19 visits to MGH) and 6 months of follow-up (up to 3 visits to MGH). Please contact the study team to obtain additional information about study visits and procedures and to review specific inclusion and exclusion criteria.

Principal Investigator: Dr. James Berry
Sponsor: Rapa Therapeutics, LLC
Enrollment Contacts: 
Kelly Fisher, 617-726-9094 or kefisher@mgh.harvard.edu 
Ann Hu, 617-724-7113 or ahu6@mgh.harvard.edu

Enrolling Studies: Clinical Research to Understand ALS

Study of ALS Sample Repository (Living Library)

Recruiting: Healthy Volunteers, Non-ALS Neuro Disease Volunteers, Motor Neuron Disease Volunteers
Full Trial Name: ALS Sample Repository
One in-person blood donation

We are developing a diverse living library of biofluid samples (blood, spinal fluid, urine) from people of different ages, ethnicities, and sexes, from healthy volunteers, people with amyotrophic lateral sclerosis (ALS), and motor neuron disease (MND), as well as other neurological diseases that may mimic motor neuron diseases. The samples collected will be stored and used for ALS research conducted globally to answer questions related to cause, prevention, treatment, and hereditability of ALS. Participants must be at least 20 years of age and be able to answer brief questions about their medical and family history, as well as be willing to have blood and/or CSF drawn for the study.

Principal Investigator: James Berry, MD, MPH
Sponsor: Project ALS, Winthrop Family Scholar in ALS Research and Clinical Care
Enrollment Contacts:
Alison Clark, 617-726-4284 or aclark51@mgh.harvard.edu
Kelly Fisher, 617-726-9094 or kefisher@mgh.harvard.edu

Study of BrainGate 2

Recruiting: People who have limited or no use of their hands, including people with ALS
Full Trial Name:
BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

Patients who have weakness due to motor neuron disease such as amyotrophic lateral sclerosis (ALS) and have no or limited use of their hands are needed for an FDA regulated research study to evaluate a new technology which may allow an individual with quadriplegia to control a computer cursor and assistive devices, like a robotic arm, by thought. This study is invasive and requires surgery. Research sessions are run at participants’ residences, so to be eligible, participants must live within 3 hours drive of Boston, MA or Providence, RI. The clinical trial requires a commitment of 13 (thirteen) months. The study is being conducted by Dr. Leigh Hochberg at Massachusetts General Hospital.

Principal Investigator: Leigh Hochberg, MD, PhD
Enrollment Contacts: clinicaltrials@braingate.org, neurotechnology@mgh.harvard.edu

Study of DIALS

Recruiting: Asymptomatic first-degree adult relatives of people with familial ALS
Full Trial Name: Dominant Inherited ALS (DIALS) Network

This study is recruiting participants who do not have any neurological symptoms, but who have a first-degree relative with ALS caused by a mutation. The purpose of the research study is to study a population at risk for developing ALS. The information collected in this study will further our understanding of underlying early disease changes to allow for development of novel therapeutics that target the earliest changes in ALS and allow for possible disease prevention.
Through this study you will be offered genetic counseling, and genetic testing for all currently known genes that may cause ALS. In addition, the study will be performing regular, longitudinal evaluations (e.g. blood samples, questionnaire completion; pulmonary and strength testing etc.,) for a period of several years.  Study visits will be completed at the Neurological Clinical Research Institute at Massachusetts General Hospital.

Principal Investigator: Katharine Nicholson, MD
Sponsor: ALS Finding a Cure, Target ALS, ALS Association, American Academy of Neurology/Muscular Dystrophy Association
Enrollment Contacts:
Kathleen Diana, 617-724-6346, kdiana@mgh.harvard.edu

Study of Fatigue in ALS

Recruiting: People with Amyotrophic Lateral Sclerosis, Healthy Volunteers
One in-person study visit

The purpose of this study is to learn if three motor tasks (walking task, upper arm task, and a fine motor hand movement task) can be used to measure fatigue in people with ALS. We are also investigating the utility of digital tools to quantify characteristics of performance fatigue. This study involves one in-person visit (lasting approximately 2 hours) where we will obtain your consent to participate in the study and ask you to complete a number of tasks, including three motor tasks designed to test performance fatigue.

During the visit, you will be asked to wear sensors that will record your movements. desired, this study can be split into two in-clinic visits occurring within 90 days of each other. Participants must be able to walk and/or use their hands, use of assistive devices is permitted.

Stipend for completion of study: $50, parking or travel reimbursement

Principal Investigator: James Berry, MD
Enrollment Contacts:
Zoe Scheier, zscheier@mgh.harvard.edu or 617-643-4803
Alison Clark, aclark51@mgh.harvard.edu or 617-726-4284

Study of Typing in ALS

Recruiting People with Amyotrophic Lateral Sclerosis, Healthy Volunteers
Enroll and participate from your home

The purpose of this study is to see if a smartphone keyboard can identify unique typing patterns in ALS and serve as a quantifiable, digital biomarker of fine motor change over time in people with ALS. This is a 9-month long study, with visits every 3 months. The visits include standard questionnaires and outcome measures, including muscle and breathing tests. The study team will help you download and install the nQ software on your smartphone. We will ask you to keep the software on your smartphone throughout the duration of the study. Ownership of a smartphone and ~15 minutes of daily use is required.

Principal Investigator:James Berry, MD, MPH
Sponsor: nQ Medical
Enrollment Contacts: Alison Clark, 617-726-4284 or aclark51@mgh.harvard.edu
Overview: https://rally.partners.org/study/typing

Study of Neuroinflammation (PBR28) Imaging

Recruiting: Patients with Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia or Frontotemporal Dementia
Healthy Volunteers Who Are Known Carriers of ALS Gene

Full Trial Name: Glial Activation Measured by PBR28-PET in People with Neurodegenerative Diseases
The purpose of the study is to learn more about inflammation in the brains of people with Motor Neuron Disease (MND) using combined Magnetic Resonance Imaging (MRI) and Positron Emission Tomography (PET). Our study will examine whether particular cells, called microglia, are hyperactive in the nervous system of people with MND, such as those individuals with ALS.

Study participation involves two visits to MGH over a maximum of three months. Participants must be between the ages of 18 and 80, be medically safe to undergo an MRI scan and be able to safely lie flat for at least 90 minutes. Additionally, participants cannot be taking any immunosuppressive medications or have a diaphragm pacing system and cannot have a diagnosis of Parkinson’s disease, Alzheimer’s disease, unstable psychiatric disease, or renal failure. All participants will be reimbursed for parking and receive compensation of $150 upon completion of each MR-PET scan. There will be additional compensation of $100 for each lumbar puncture completed by individuals with MND.

Principal Investigator:
 Suma Babu, MD, MPH

Sponsors: Neurodegenerative Disease Pilot Study Grant, K23 NS 083715, Evan and Arlene Yegelwel Endowed Fund for Primary Lateral Sclerosis Research and Care, PET Imaging of inflammation and epigenetics in ALS (ALS ONE), Muscular Dystrophy Association, Sundry

Enrollment Contacts:
Austin Lewis, alewis29@mgh.harvard.edu or  617-724-7928
Mackenzie Keegan, mkeegan@mgh.harvard.edu or 617-643-6252

Study of Nutrition Using mHealth App

Enroll in this study from your home

Researchers at Massachusetts General Hospital have designed an app to calculate and track ALS calorie goals. Studies show that people with ALS do better when they maintain their weight, compared to those who lose weight.

We would like to provide further information from our previous announcement on how to download our ALS Nutrition App. There are TWO apps that will need to be downloaded:

  1. ALS Nutrition App: our nutrition app which we made specifically for ALS.
  2. A diet diary to track your dietary intake. We recommend the Bitesnap diet diary which will automatically synch your information with the ALS Nutrition App.

To participate, first install the MyDataHelps app from the Google Play Store or Apple Store. Then, open it and search for "ALS Nutrition" under "Join Studies."

Then, install Bitesnap from the Google Play Store or Apple Store.

Principal Investigator: Anne-Marie Wills, MD

Enrollment Contact: Mansi Sharma, mghALSapp@partners.org or 617-643-2400.

You can also visit us at projects.iq.harvard.edu/alsnutrition.

Study of Radicava Effects in ALS

Full Trial Name: Radicava/(Edaravone) Findings in Biomarkers in ALS (REFINE-ALS)
Enroll & participate from home!

Radicava has been shown to slow the loss of physical function in ALS and was approved by the FDA as a treatment for ALS in 2017. The purpose of this observational study is to provide a deeper understanding of the biological effects of Radicava in participants with ALS. REFINE-ALS will measure the levels of distinct biomarkers involved in oxidative stress and in inflammatory response, neuronal injury or death, and muscle injury.

All participants must make the clinical decision to be prescribed Radicava prior to enrolling and screening for the study. Participants will be followed over six cycles of Radicava as an intravenous (IV) infusion over 24 weeks, with blood and urine samples collected at each visit for analyses. Biomarker levels and ALS progression will be assessed before initiating treatment, at the start of treatment, and at specific times throughout the study. The study requires 8 study visits to MGH over approximately 6 months.

Principal Investigator: Suma Babu, MD
Sponsor: MT Pharma
Enrollment Contacts:
Austin Lewis, 617-724-7928 or alewis29@mgh.harvard.edu
Mackenzie Keegan, 617-643-6252 or mkeegan@mgh.harvard.edu

Study of Skin Biopsy/Stem Cells for Research in MND

Recruiting: Patients with Amyotrophic Lateral Sclerosis or Primary Lateral Sclerosis, healthy volunteers
Full Trial Name: Stem Cells for Research in Motor Neuron Diseases (MND)

Neurodegenerative diseases are diseases in which nerve cells of the brain and spinal cord die. There is a need to understand the cause of these diseases and to develop treatments. Recent advancements in stem cell technology have allowed us to create a person’s own nerve cells by taking a skin biopsy or blood sample. This study wants to use this new technology to make models for neurodegenerative diseases. We hope this will give us a better understanding of the diseases, enable us to use the cells for drug screening, and in the future, develop treatments.

Principal Investigator: James Berry, MD, MPH
Sponsor: Harvard Stem Cell Institute
Enrollment Contacts:
Alison Clark, 617-726-4284 or aclark51@mgh.harvard.edu

Study of Smartphone App for Symptom Monitoring

Enroll and participate in this study from your home!

Recruiting: Patients with Amyotrophic Lateral Sclerosis
Full Trial Name: Feasibility and Sensitivity of a Symptom Monitoring Application in Real Time (SMART) for Amyotrophic Lateral Sclerosis

The study asks each participant to use the smartphone application for a few minutes every day by answering a questionnaire/survey, recording your voice and/or performing an on-screen exercise. The purpose of the research study is to determine how helpful a smartphone application would be in collecting research data and to learn more about disease progression. This is a pilot study in which a smartphone application was designed and customized for use in ALS clinical trials.

Individuals with ALS will be in the study for about 12 months.

The study is currently recruiting participants who meet the following guideline: Adults with Amyotrophic Lateral Sclerosis (ALS) to download and use the smartphone application using their smartphone device running iOS 8 or higher, or Android 4.1 or higher.

Principal Investigator: James Berry MD, MPH
Sponsor: ALS Finding a Cure
Enrollment Contacts:
Zoe Scheier, zscheier@mgh.harvard.edu or 617-643-4803
Alison Clark, aclark51@mgh.harvard.edu or 617-726-4284

Study of Speech Motor Impairment in ALS

Full Trial Name: Speech Motor Impairments: Coordination of tongue, lips, and jaw
Enroll and participate in this trial from your home

The Speech and Feeding Disorders Lab at MGH Institute of Health Professions is interested in studying the movements the face and mouth during speech, chewing and swallowing in persons diagnosed with ALS. You will be asked to fill out a health questionnaire and repeat various sounds and sentences while the movements of your face and mouth are recorded. Study sessions can be completed remotely using your own computer or device. This research aims to help improve the diagnosis and treatments of ALS.

Principal Investigator:
 Jordan Green, Ph.D.
Sponsors: National Institutes of Health and the American Speech-Language-Hearing Foundation
Enrollment Contact: Speech and Feeding Disorders Lab, 617-724-6347 or speechfeedinglab@mghihp.edu

No Longer Enrolling

Trial of BIIB078 for C9ORF72-ALS

Full Trial Name: A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults with C9ORF72-Associated Amyotrophic Lateral Sclerosis
Trial Phase: 1
Principal Investigator: Suma Babu, MD, MPH
Sponsor: Biogen MA Inc.

We are doing this research study to find out about the safety and tolerability of the study drug BIIB078. The study is funded by Biogen MA Inc. This study is recruiting patients with C9ORF72-Associated Amyotrophic Lateral Sclerosis (ALS) with a slow vital capacity greater than or equal to 50% of predicted value. Participation in the study will last for approximately 40 weeks and will include an overnight stay at MGH in addition to in person visits. The study team can provide additional information on the number of required visits during your initial visit. There are additional inclusion/exclusion criteria that the study team will review with you in more detail if you are interested in participating. For more information regarding this trial, or to further discuss how to participate, please contact the following study coordinators:

Gabriel Jacobs, 617-726-3015 or gjacobs@mgh.harvard.edu
Kush Mehta, 617-643-5376 or kmehta9@mgh.harvard.edu 
Ryan Gifford, 617-724-0783 or rfgifford@mgh.harvard.edu
Shea Golden, 617-724-3268 or sgolden3@mgh.harvard.edu

Trial of BIIB100 for ALS

Full Trial Name: Double-Blind, Placebo-Controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adult Participants With ALS
Trial Phase: 1

We are doing this Phase I research study to find out about the safety and tolerability of the study drug BIIB100. BIIB100 is taken orally. This study is placebo-controlled, which means that some participants will receive study medication that contain no BIIB100.

The study is recruiting for patients with a diagnosis of Amyotrophic Lateral Sclerosis who have a slow vital capacity of >65%.

Participation in the study will last for 6 weeks and will include a 2-night overnight stay at MGH in addition to 2-3 in-person visits. During the overnight stay, you will receive a single dose of the study medication.

There are additional inclusion/exclusion criteria that the study team will review with you in more detail if you are interested in participating. For more information regarding this trial, or to further discuss how to participate, please contact one of the study coordinators.

Principal Investigator: Dr. Katharine Nicholson.
Sponsor: Biogen MA, Inc.
Enrollment Contacts: Judi Carey, 617-724-8995 or jcarey8@partners.org

Trial of BIIB067 for SOD1-ALS

Full Trial Name: A Phase 1, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerosis
Trial Phase: 3

We are doing this research study to find out about the safety and tolerability of the study drug BIIB067. The study is funded by Biogen MA Inc. This study is recruiting patients with SOD1-Amyotrophic Lateral Sclerosis (SOD1-ALS) with a forced vital capacity greater than or equal to 50% of predicted value. Participation in the study will last for approximately 31 weeks and will include an overnight stay at MGH in addition to in person visits. The study team can provide additional information on the number of required visits during your initial visit. There are additional inclusion/exclusion criteria that the study team will review with you in more detail.

Principal Investigator: Suma Babu, MD, MPH
Sponsor: Biogen MA Inc.
Enrollment Contact:
Gabe Jacobs, 617-726-3015 or gjacobs@mgh.harvard.edu
Kush Mehta, 617-643-5376 or kmehta9@mgh.harvard.edu
Ryan Gifford, 617-724-0783 or rfgifford@mgh.harvard.edu
Shea Golden, 617-724-3268, sgolden3@mgh.harvard.edu