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

We are doing this research study to find out about the safety and tolerability of the study drug AT-1501. This study is recruiting Visit the MGH Healey Center Website: patients diagnosed for 24 months or less with Amyotrophic Lateral Sclerosis (ALS) with n http://www.massgeneral.org/als/ ALSFRS-R score of 35 or higher. Participation in this study will last for 19 Then select Research Opportunities from weeks and will include up to 7 visits to the dropdown menu to read information Massachusetts General Hospital. about ALS trials and studies. There are additional inclusion/exclusion criteria that the study team will review with To receive eblasts about the Platform Trial, Interventional Trials you in more detail if you are interested in other NEALS ALS trials, and, clinical care participating. For more information regarding at MGH, sign up for the Link: this trial, or to further discuss how to Currently Enrolling participate, please contact one of the Sign up for ALS Link following study coordinators. For more information regarding this trial, or to To read updates about the Healey Platform further discuss how to participate, please Trial, select Platform Trial from the contact one of the study coordinators. dropdown menu on opening screen. Principal Investigator: Sabrina Paganoni, MD, PhD. Sponsor: Anelixis Therapeutics Inc. Enrollment Contact: Zoe Scheier, 617-643- 4803, [email protected]

For more information about these trials: Contact the research coordinator listed for trial(s) you are interested in OR Judi Carey, Research Access Nurse, at [email protected] or 617-724-8995

Updated: September 2021

Trial of BLZ945 for ALS Trial of BIIB105 for ALS and polyQ-ALS Trial of RAPA-501 Cell Therapy for ALS Full Trial Name: Open-label, adaptive design Full Trial Name: A Phase 1 Multiple-Ascending- Full Trial Name: Phase I Trial of Autologous study in patients with ALS to characterize safety, Dose Study to Assess the Safety, Tolerability, and Hybrid TREG/Th2 Cell Therapy (RAPA-501) for tolerability and microglia response, as Pharmacokinetics of BIIB105 Administered Amyotrophic Lateral Sclerosis measured by TSPO binding, following multiple Intrathecally to Adults with Amyotrophic Lateral Trial Phase: 1 Sclerosis With or Without Poly-CAG Expansion in doses of BLZ945 using positron emission We are doing this Phase I, three-cohort research tomography (PET) with radioligand [11C]- the Ataxin-2 Gene PBR28 Trial Phase: 1 study to find out if RAPA-501 cell therapy is safe in Trial Phase: 2 The purpose of this Phase 1 research study is to learn patients with ALS. This is an open label study, which This research study is being done to study the about the safety and tolerability of the study drug means that there is no placebo, and all participants safety and tolerability of a molecule called BIIB105 in adults with a diagnosis of Amyotrophic will receive actual RAPA-501 cell infusions. Two BLZ945 in patients with ALS. We also want to Lateral Sclerosis who have a slow vital capacity of doses of RAPA-501 cells will be investigated for find out if BLZ945 is safe to take without causing safety. In addition, if RAPA-501 cells are found to too many side effects in ALS. Novartis is the ≥60%. This study will also look at the level and be safe, additional patients will receive RAPA-501 sponsor of this study, and BLZ945 is taken orally. action of the study drug in your body, and what This research study will use an imaging method happens to this level over time. BIIB105 is cells in combination with a low dose of two known as Positron Emission Tomography or PET administered intrathecally (via lumbar puncture into chemotherapy agents. The study is looking for to measure the effect of BLZ945 on a specific the lower spine). This study is placebo-controlled, patients with a diagnosis of Amyotrophic Lateral inflammatory cell type in the brain called which means that some participants will receive Sclerosis who have a forced vital capacity of ≥ 50%. microglia, which are activated in ALS. The study placebo, which looks like the study drug but does not In this study, participants’ white blood cells (T-cells) aims at providing important information on will be removed during a procedure called Apheresis whether BLZ945 could be a potential treatment contain any active ingredients. (a type of blood filtering). The T cells will be made for patients with ALS and to help to select the Participation in the study will last for approximately most appropriate doses for the planning of future 29 weeks, including a 4-week screening period, 13- into the specialized RAPA-501 cells and re-infused research in patients with ALS. This study is open week treatment period and a 12-week follow-up through an I.V. Total study participation will last label, which means that all participants receive period. During the treatment period, participants will about 1 year; 6 months of treatment (up to 19 visits study drug. Study participation will last, at most, receive 3 loading doses of BIIB105 every 2 weeks, to MGH) and 6 months of follow-up (up to 3 visits to 70 days and include up to 5-7 hospital admission followed by 2 doses administered once every 4 MGH). Please contact the study team to obtain days at MGH. Please contact the study team to additional information about study visits and obtain additional information. weeks, for a total of 5 doses. For more information, Principal Investigator: Suma Babu, MD, MPH please contact one of the coordinators. procedures and to review specific inclusion and Sponsor: Novartis Principal Investigator: Dr. Suma Babu exclusion criteria. Enrollment Contact: Sponsor: Biogen MA Inc. Principal Investigator: Dr. James Berry Austin Lewis, 617-724-7928, Enrollment Contact: Mackenzie Keegan, 617-643- Sponsor: Rapa Therapeutics, LLC [email protected]; Mackenzie Keegan, Enrollment Contact: Kelly Fisher, 617-726- [email protected], 617-643-6252 6252, [email protected]; or Gabriel Jacobs, 617-726-3015, [email protected] 9094, [email protected] or Ann Hu, 617-724-7113, [email protected]

Stay Connected to the Platform Trial More investigational products are anticipated to be added to the HEALEY ALS Platform Trial through support by pharma, foundation partners, philanthropy, federal, and other fundraising initiatives.

Highlights of the HEALEY Visit our website to learn more about current and future regimens: ALS Platform Trial www.massgeneral.org/neurology/als/research/first- platform-trial-treatments HEALEY ALS • This trial is being conducted at 50+ sites Register to attend weekly Platform Trial updates nationwide, with more US sites expected to join. or view recordings of previous webinars: https://www.massgeneral.org/neurology/als/research/ Platform Trial: • Participation in the trial lasts approximately 6 platform-trial-news months and involves about 7 in-person visits. Sign up for the ALS Link to receive news and updates about research and clinical care from the • Approximately 160 participants will be enrolled in Healey Center for ALS at Mass General: Overview and Currently each regimen. The trial is perpetual, so enrollment https://lp.constantcontactpages.com/su/saTzwIp/ALS will continue as more investigational products are Link Enrolling Regimens added over time. For general questions about the HEALEY ALS • The active study drug to placebo ratio is 3:1 across Platform Trial, contact the Patient Navigator: all regimens (meaning there is a 75% chance of email: [email protected] receiving active drug, 25% chance of placebo). phone: 833-425-8257 (HALT ALS)

• Eligible participants who provide informed consent Stay Connected to ALS to the master protocol will be randomly assigned to one of the available regimens. Research Worldwide

• After completing a regimen, participants may have To learn about investigational drug trials or the option to receive the investigational product observational studies for ALS, please visit: through an open label extension (100% chance of www.clinicaltrials.gov www.neals.org receiving active drug). www.iamals.org

www.als.net • If you are interested in participating, the study team

will review the inclusion/exclusion criteria with you Register for the National ALS Registry: Updated April 2021 in more detail. www.cdc.gov/als/ALSJoinALSRegistry.html

What is a Platform Trial? How does the Platform Trial work? Current Platform A platform trial is a trial in which multiple Participants in the Platform Trial will be randomly investigational products are tested at the same time in assigned to one of the regimens that are available at Regimens different participants using a master protocol and the time of their enrollment. Each participant will then Each regimen in the trial is designed to test whether specialized statistical tools. This results in a more be randomized within a regimen to receive active drug an investigational product is safe and effective in efficient and expedited trial. More regimens can be or placebo. All current regimens in the Platform Trial people diagnosed with ALS. added as new investigational products become have a 3:1 active study drug to placebo ratio. available; thereby decreasing or eliminating the gap in Regimen A: Trial of Zilucoplan time from identification of a potential therapy to trial What is a Regimen? Developed by UCB in humans. A regimen is an arm of the trial that specifies the Zilucoplan is designed to work by blocking a dosage, schedule, and duration of experimental protein called complement component 5 (C5), treatment with an investigational product. After which may lead to a reduction in tissue damage informed consent to the master protocol, each participant will be randomly assigned to one regimen. caused by the immune system in ALS. This drug All regimens follow the Master Protocol but may is administered as a daily injection under the include additional activities and inclusion/exclusion skin. criteria. Regimen B: Trial of Verdiperstat Developed by Biohaven Pharmaceuticals Platform Trial Contact Verdiperstat is an investigational anti- inflammatory drug that inhibits myeloperoxidase Information at Mass and may work by reducing neural inflammation Why Platform Trial? General Hospital that occurs in ALS. This drug is administered as Faster answers, More access, Less placebo, More two pills taken by mouth twice a day. learning about ALS. Regimen C: Trial of CNM-Au8 Platform trials are designed to decrease the amount of If interested in participating in the Developed by Clene Nanomedicine Platform Trial at the Mass General site, time it will take to find successful therapies; improve CNM-Au8 is a liquid suspension of pure gold study medication to placebo ratio; and increase access please fill out our Patient Interest Form online at: nanocrystals that may work by providing an to participants by conducting the same trial at multiple https://redcap.partners.org/redcap/surveys/? energetic assist to impaired motor and research centers. The platform trial is perpetually open s=DX8H3CXWTH improving their ability to function. This drug is until successful treatments are discovered. Our study staff will reach out to you with taken daily as a 2oz drink.

further details about the Platform Trial after Regimen D: Trial of Pridopidine receiving your completed Patient Interest Developed by Prilenia Therapeutics Form. Principal Investigator: Sabrina Paganoni, MD Pridopidine is a selective and potent activator of Study Coordinator: Danny Hevert, Mia a protein called the Sigma-1 receptor (S1R). The Resendes, Simon Shulman S1R is highly expressed in the brain and spinal Email: [email protected] cord. Activation of the S1R promotes brain cell Phone: 617-643-3902 health, function and prevents neuronal damage in

ALS. Pridopidine is a capsule taken by mouth twice daily.

Stay Connected to the Platform Trial More investigational products are anticipated to be added to the HEALEY ALS Platform Trial through support by pharma, foundation partners, philanthropy, federal, and other fundraising initiatives.

Visit our website to learn more about current and

future regimens: www.massgeneral.org/neurology/als/research/first- HEALEY ALS platform-trial-treatments Register to attend weekly Platform Trial updates or view recordings of previous webinars: Platform Trial: https://www.massgeneral.org/neurology/als/research/ platform-trial-news Current Regimens Sign up for the ALS Link to receive news and updates about research and clinical care from the Healey Center for ALS at Mass General: https://lp.constantcontactpages.com/su/saTzwIp/ALS Link

For general questions about the HEALEY ALS Platform Trial, contact the Patient Navigator: email: [email protected] phone: 833-425-8257 (HALT ALS)

Stay Connected to ALS Research Worldwide Thank you for your interest in the HEALEY ALS To learn about investigational drug trials or Platform Trial. This brochure provides a brief observational studies for ALS, please visit: overview of the currently enrolling regimens. Upon www.clinicaltrials.gov enrolling in the trial, participants will be given www.neals.org additional detailed information about the regimen to www.iamals.org which they are assigned. www.als.net Updated April 2021 Register for the National ALS Registry: www.cdc.gov/als/ALSJoinALSRegistry.html

What is a Regimen? of inflammation seen in ALS) in previous clinical A regimen is an arm of the trial that specifies the trials for Parkinson’s disease. dosage, schedule, and duration of experimental Regimen C: Trial of CNM-Au8 treatment with an investigational product. All Developed by Clene Nanomedicine regimens in the Platform Trial involve about 7 in- CNM-Au8 may provide an energetic assist to person visits over the course of 6 months. Participants impaired motor neurons and help improve their are randomly assigned to one the regimens (meaning ability to function normally. Each 2 oz dose of neither the researchers nor the participant can choose CNM-Au8 is a concentrated, liquid suspension of the regimen), then randomly assigned to active drug or placebo. There is a 75% chance of receiving active Current Platform pure gold nanocrystals that study participants drug and a 25% chance of receiving placebo. After Regimens drink every morning. These extremely small completing the 6-month trial, participants can choose nanocrystals travel through the body and enter Please discuss the potential benefits and risks of to join the open label extension for their regimen with the brain and motor cells where they may each investigational product and any concerns a 100% chance of receiving the active drug. enhance the ability of motor neurons to survive you may have with your study physician. and communicate by supporting cellular Regimen A: Trial of Zilucoplan metabolism. Side effects may include headache, What is an Open Label Extension? Developed by UCB Most regimens in the Platform Trial will offer an dizziness, diarrhea, and fatigue. Previously, Zilucoplan blocks a protein called complement Open Label Extension (OLE). Participants who CNM-Au8 was demonstrated to be safe and well- component 5 (C5), which may lead to a reduction complete the 6-month trial may be eligible to receive tolerated by healthy volunteers in a Phase 1 active drug during the OLE. The OLE will only be in tissue damage in ALS that is carried out by the study. available for the drug being tested in the regimen to immune system. Participants and their caregivers Regimen D: Trial of Pridopidine will be trained on how to safely inject a prefilled which the participant was assigned. Duration of OLE Developed by Prilenia Therapeutics syringe of Zilucoplan under the participant’s skin may vary by regimen. Pridopidine selectively binds and activates the daily. Because this drug affects the immune Sigma-1 receptor (S1R), a protein highly system, vaccination against meningitis is How are drugs selected in this trial? expressed in the brain and spinal cord. By required before starting this regimen. Side effects Investigational products included in the Platform Trial activating the S1R, pridopidine enhances several have been selected by a team of experts (the Therapy of zilucoplan may include bruising, redness, cellular pathways that are disrupted in ALS and Evaluation Committee) after careful review of the pain, discomfort, itching, a lump or scabbing at thus may improve a neuron’s function and health. company and the science behind the medication. the injection site. Previous trials in other diseases Pridopidine is the first drug to show a beneficial Criteria for selection have included robust preclinical indicated that Zilucoplan is safe and tolerable. data (data from the lab that support the scientific effect in maintaining function (activities of daily rationale for testing these drugs in ALS) and previous Regimen B: Trial of Verdiperstat living) of Huntington Disease patients in a human experience in ALS or other neurological Developed by Biohaven Pharmaceuticals, Inc. clinical trial. Pridopidine is a capsule taken twice diseases (to support the dose, safety, and the ability of Verdiperstat is an investigational anti- daily. An extensive safety data base of each drug to target the intended mechanism in the inflammatory drug that inhibits myeloperoxidase approximately 1000 subjects shows that body). Each regimen has an equal chance of success and may reduce oxidative stress and pridopidine 45 mg twice daily has a safety profile based on scientific evidence reviewed by the Therapy inflammation levels in the brain and spinal cord. similar to placebo. Patients with a significant Evaluation Committee. This drug is administered as two pills taken by heart condition or those taking high doses of mouth twice a day. Verdiperstat has shown the Nuedexta, Citalopram, or Escitalopram may not potential to reduce microglial activation (a kind be eligible for this regimen.

Study of Radicava Effects in ALS Study of Skin Biopsy/Stem Cells for Research +Amyotrophic Lateral Sclerosis in MND Full Trial Name: Radicava/(Edaravone) +ALS +PLS +Healthy Volunteers Enroll & participate in study from your home! Full Trial Name: Stem Cells for Research in Findings in Biomarkers in ALS (REFINE-ALS) Motor Neuron Diseases (MND) Radicava has been shown to slow the loss of Neurodegenerative diseases are diseases in physical function in ALS and was approved by which nerve cells of the brain and spinal cord the FDA as a treatment for ALS in 2017. The die. There is a need to understand the cause of purpose of this observational study is to provide these diseases and to develop treatments. Recent a deeper understanding of the biological effects advancements in stem cell technology have of Radicava in participants with ALS. REFINE- allowed us to create a person’s own nerve cells ALS will measure the levels of distinct by taking a skin biopsy or blood sample. This biomarkers involved in oxidative stress and in study wants to use this new technology to make inflammatory response, neuronal injury or models for neurodegenerative diseases. We death, and muscle injury. hope this will give us a better understanding of Biofluid Biomarker & All participants must make the clinical decision the diseases, enable us to use the cells for drug to be prescribed Radicava prior to enrolling and screening, and in the future, develop treatments. Observational Studies screening for the study. Participants will be Principal Investigator: James Berry, MD, MPH followed over six cycles of Radicava as an Sponsor: Harvard Stem Cell Institute Currently Enrolling intravenous (IV) infusion over 24 weeks, with Enrollment Contact: Alison, Clark, 617-726- blood and urine samples collected at each visit 4284, [email protected] 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 Visit the MGH Healey Center Website: requires 8 study visits to MGH over http://www.massgeneral.org/als/ approximately 6 months. Principal Investigator: Suma Babu, MD Then select Research Opportunities from Sponsor: MT Pharma the dropdown menu to read information

Enrollment Contact: Austin Lewis, 617-724- about ALS trials and studies. 7928, [email protected]; Mackenzie

Keegan, 617-643-6252, To receive eblasts about the Platform Trial, [email protected] other NEALS ALS trials, and, clinical care

at MGH, sign up for the Link:

Sign up for ALS Link

For more information about these trials:

Contact the research coordinator listed for To read updates about the Healey Platform trial(s) you are interested in OR Judi Carey, Trial, select Platform Trial from the Research Access Nurse, at dropdown menu on opening screen. [email protected] or 617-724-8995 Updated: September 2021

Study of DIALS Study of ALS Sample Repository (Living + Asymptomatic first-degree adult relatives of Library) people with familial ALS +Amyotrophic Lateral Sclerosis Full Trial Name: Dominant Inherited ALS +Healthy Volunteers (DIALS) Network +Non-ALS Neuro Disease Volunteers

This study is recruiting participants who do not +Motor Neuron Disease Volunteers have any neurological symptoms, but who have One In-Person Blood Collection a first-degree relative with ALS caused by a Full Trial Name: ALS Sample Repository mutation. The purpose of the research study is We are developing a diverse living library of to study a population at risk for developing biofluid samples (blood, spinal fluid, urine) ALS. The information collected in this study from people of different ages, ethnicities, and will further our understanding of underlying sexes, from healthy volunteers, people with early disease changes to allow for development amyotrophic lateral sclerosis (ALS), and motor of novel therapeutics that target the earliest neuron disease (MND), as well as other changes in ALS and allow for possible disease neurological diseases that may mimic motor prevention. neuron diseases. Samples collected will be Through this study you will be offered genetic stored and used for ALS research conducted counseling, and genetic testing for all currently globally to answer questions related to cause, known genes that may cause ALS. In addition, prevention, treatment, and hereditability of the study will be performing regular, ALS. Participants must be at least 20 years old longitudinal evaluations (e.g. blood samples, and be able to answer brief questions about their questionnaire completion; pulmonary and medical and family history, as well as be willing strength testing etc.,) for a period of several to have blood and/or CSF drawn for the study. years. Study visits will be completed at the Principal Investigator: James Berry, MD, MPH Neurological Clinical Research Institute at Sponsor: Hollister Lindley Fund

Massachusetts General Hospital. Enrollment Contact:

Principal Investigator: Katharine Nicholson, MD Alison, Clark, 617-726-4284 , Sponsor: ALS Finding a Cure, Target ALS, [email protected], or, Kelly Fisher, ALS Association, American Academy of 617-726-9094, [email protected] Neurology/Muscular Dystrophy Association

Enrollment Contact: Kathleen Diana, 617-

724-6346, [email protected]

Visit the MGH Healey Center Website: http://www.massgeneral.org/als/

Then select Research Opportunities from the dropdown menu to read information about ALS trials and studies.

To receive eblasts about the Platform Trial, other ALS trials, and, clinical care at MGH, sign up for the Link:

Sign up for ALS Link Imaging

To read updates about the Healey Platform Trial, select Platform Trial from the Observational Studies dropdown menu on opening screen. Currently Enrolling

For more information about these trials:

Contact the research coordinator listed for trial(s) you are interested in OR Judi Carey, Research Access Nurse, at [email protected] or 617-724-8995

Updated: September 2021

Study of SPINE-ALS Neuroinflammation (PBR28) Imaging Study +Amyotrophic Lateral Sclerosis +Amyotrophic Lateral Sclerosis (<18 mo. from symptom +Healthy Volunteers onset) Full Trial Name: Positron Emission +Primary Lateral Sclerosis Tomography to Characterize in vivo +Hereditary Spastic Paraplegia Neuroinflammation in the Spinal Cord in People +Healthy Volunteers (known carriers of ALS gene) with ALS +Frontotemporal Dementia We are doing this research to learn more about Full Trial Name: Glial Activation Measured by changes in the spinal cord and brain in ALS. PBR28-PET in People with Neurodegenerative “Microglia” are a type of immune cell that we Diseases are particularly interested in. We would like to The purpose of the study is to learn more about find out if microglia are activated in the spinal inflammation in the of people with Motor cord and brain of individuals with ALS. Special Neuron Disease (MND) using combined Magnetic imaging techniques are now available to test for Resonance Imaging (MRI) and Positron Emission changes in microglia. Magnetic Resonance Tomography (PET). Our study will examine Imaging (MRI) and Positron Emission whether particular cells, called microglia, are Tomography (PET) are two tests that allow us to hyperactive in the nervous system of people with take pictures and “look inside” the body without MND, such as those individuals with ALS. surgery. MR-PET scanners use both MRI and Study participation involves two visits to MGH PET tests at the same time. The MR-PET over a maximum of three months. Participants must scanner may give clearer images and more be between the ages of 18 and 80, be medically safe information about the inside of the body. to undergo an MRI scan and be able to safely lie If you choose to take part in this study you may flat for at least 90 minutes. Additionally, have 5 visits at MGH, up to 3 months apart. We participants cannot be taking any will pay you $150 for completion of the spinal immunosuppressive medications or have a cord MR-PET scan. If you choose to participate diaphragm pacing system and cannot have a in the optional brain MR-PET scan you will be diagnosis of Parkinson’s disease, Alzheimer’s paid an additional $50 for completion. disease, unstable psychiatric disease, or renal Principal Investigators: Suma Babu, MD, MPH failure. All participants will be reimbursed for Sponsors: ALS Finding a Cure Foundation, Voyager parking and receive compensation of $150 upon Therapeutics completion of each MR-PET scan. There will be Enrollment Contacts: additional compensation of $100 for each lumbar Austin Lewis, 617-724-7928, puncture completed by individuals with MND. [email protected], or, Mackenzie Keegan, Principal Investigator: Suma Babu, MD, MPH 617-643-6252, [email protected] 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, 617-724-7928, [email protected], or, Mackenzie Keegan,

617-643-6252, [email protected]

Study of Nutrition using mHealth App Study of Smartphone App for ALS +Amyotrophic Lateral Sclerosis +Amyotrophic Lateral Sclerosis Full Trial Name: The E-health Application To Enroll & participate in study from your home Modify Oral Energy Intake & Measure Full Trial Name: Feasibility and Sensitivity of Enroll & participate in study from your home a Symptom Monitoring Application in Real Outcomes Remotely in ALS (EAT MORE 2) Time (SMART) for ALS Researchers at MGH have designed a mobile The study asks each participant to use the health app to help people with ALS calculate smartphone application for a few minutes every and track their ideal calories. The app is based day by answering a questionnaire/survey, upon prior MGH research which demonstrated recording your voice and/or performing an on- that maintaining weight appeared to improve screen exercise. The purpose of the study is to ALS disease progression and quality of life. determine the usefulness of a smartphone app in The new ALS Nutrition app provides nutritional collecting research data and to learn more about counseling tailored to individual needs, disease progression. Individuals with ALS will monitors weight, provides recommendations be participating for about 12 months. about how much and what foods to eat, and The study is currently recruiting participants contains over 100 recipes created by the who meet the following: Adults with Digital Observational Registered Dietitians at MGH. People who Amyotrophic Lateral Sclerosis (ALS) to download the app can use the app for free and download and use the smartphone application Studies Currently they also have an option to share their data with using their smartphone device running iOS 8 or MGH researchers. higher, or Android 4.1 or higher. To access the app, download the CareEvolution Principal Investigator: James Berry MD, MPH Enrolling platform & search for the ALS Nutrition study: Sponsor: ALS Finding a Cure • Android: https://play.google.com/st Enrollment Contact: Zoe Scheier, [email protected], 617-724-4663; ore/apps/details?id=com.careevoluti on.mydatahelps Alison, Clark, [email protected], 617- 726-4284 • AppleiPhone: https://apps.apple.com /app/mydatahelps/id1286789190 Visit the MGH Healey Center Website: Principal Investigator: Anne-Marie Wills MD, http://www.massgeneral.org/als/ MPH Sponsor: ALS Association Then select Research Opportunities from Enrollment Contact: Mansi Sharma, 617-643- the dropdown menu to read information 2400, [email protected] about ALS trials and studies. Or visit us at: https://projects.iq.harvard.edu/alsnutrition To receive eblasts about the Platform Trial, other ALS trials, and, clinical care at MGH,

sign up for the Link: For more information about these trials:

Sign up for ALS Link Contact the research coordinator listed for trial(s) you are interested in OR Judi Carey, Research Access Nurse, at To read updates about the Healey Platform

[email protected] or 617-724-8995 Trial, select Platform Trial from the

dropdown menu on opening screen. Updated: September 2021

Study of Fatigue in ALS Study of Speech Motor Impairment in ALS Study of Typing in ALS +ALS, +Healthy Volunteers +Amyotrophic Lateral Sclerosis +Amyotrophic Lateral Sclerosis One In-Person Study Visit Enroll and participate from your home +Healthy Volunteers The purpose of this study is to learn if three Full Trial Name: Speech Motor Impairments: Enroll and participate from your home motor tasks (walking task, upper arm task, and a Coordination of tongue, lips, and Jaw The purpose of this study is to see if a smartphone fine motor hand movement task) can be used to The Speech and Feeding Disorders Lab at MGH keyboard can identify unique typing patterns in measure fatigue in people with ALS. We are Institute of Health Professions is interested in ALS and serve as a quantifiable, digital biomarker also investigating the utility of digital tools to studying the movements the face and mouth of fine motor change over time in people with ALS. quantify characteristics of performance fatigue. during speech, chewing and swallowing in persons This is a 9-month long study, with visits every 3 This study involves one in-person visit (lasting diagnosed with ALS. You will be asked to fill out months. The visits include standard questionnaires approx. 2 hours) where we will obtain your a health questionnaire and repeat various sounds and outcome measures, including muscle and and sentences while the movements of your face breathing tests. The study team will help you consent to participate in the study and ask you and mouth are recorded. Study sessions can be download and install the nQ software on your to complete a number of tasks, including three completed remotely using your own computer or smartphone. We will ask you to keep the software motor tasks designed to test performance device. This research aims to help improve the on your smartphone throughout the duration of the fatigue. During the visit, you will be asked to diagnosis and treatments of ALS. study. Ownership of a smartphone and ~15 minutes wear sensors that will record your movements. Principal Investigator: Jordan Green, Ph.D. of daily use is required. desired, this study can be split into two in-clinic Sponsors: National Institutes of Health and the Principal Investigator: James Berry, MD, MPH visits occurring within 90 days of each other. American Speech-Language-Hearing Foundation Sponsor: nQ Medical Participants must be able to walk and/or use Enrollment Contact: Speech and Feeding Enrollment Contacts: Alison, Clark, 617-726- their hands, use of assistive devices is permitted. Disorders Lab 617-724-6347, 4284, [email protected] Stipend for completion of study: $50, parking or [email protected] Overview: https://rally.partners.org/study/typing travel reimbursement Principal Investigator: James Berry, MD Study of BrainGate 2 Enrollment Contacts: Zoe Scheier, +Amyotrophic Lateral Sclerosis [email protected], 617-643-4803; Full Trial Name: BrainGate2: Feasibility Study Alison, Clark, 617-726-4284, of an Intracortical Neural Interface System for [email protected] 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

months. The study is being conducted by Dr. Leigh Hochberg at Mass General Hospital.

Principal Investigator: Leigh Hochberg, M.D.,

Ph.D.

Enrollment Contact: [email protected], @mgh.harvard.edu