Clinical Research at the Healey & AMG Center for ALS

The HEALEY Platform Trial is a perpetual adaptive trial. Future regimens will be added as a new study drugs become available. Register for the Weekly Platform Trial Q&A Webinars to stay connected to trial news and updates.  

Learn more about the platform trial.

Current Active Regimens

Regimen F: Trial of ABBV-CLS-762 (No Longer Enrolling, Enrollment Goals Met)
Developed by Calico in collaboration with AbbVie, ABBV-CLS-7262 targets eIF2B, a key regulator of the integrated stress response (ISR), a pathway activated in people with ALS. In neurons exposed to cellular stressors, inhibition of the ISR by ABBV-CLS-7262 restores protein synthesis and dissolves pre-formed TDP-43 containing stress granules. This effect of ABBV-CLS-7262 is of clinical interest because TDP-43 containing stress granules are thought to lead to TDP-43 inclusions, a hallmark of ALS pathology.

Regimen G: Trial of DNL343 (No Longer Enrolling, Enrollment Goals Met)
DNL343 is a novel investigational ALS therapy that targets eIF2B, a central regulator of the integrated stress response (ISR). The ISR appears to be overactive in ALS, leading to the formation of stress granules containing TDP-43. Buildup of TDP-43 is harmful and leads to neuronal degeneration. In the lab, inhibition of the ISR by DNL343 dissolves TDP-43 containing stress granules and decreases ISR biomarkers. The safety, pharmacokinetics, and pharmacodynamics of DNL343 have been characterized in both healthy participants and people with ALS, in a Phase 1 (N=47) and a Phase 1b (N=29) study, respectively, with dosing for up to 28 days. Results from both studies demonstrated that once-daily oral dosing with DNL343 was generally well tolerated and exhibited extensive Cerebrospinal Fluid (CSF) penetration. In addition, robust inhibition of biomarkers associated with the ISR pathway was observed in blood samples from study participants.

No Longer Enrolling

Regimen A: Trial of Zilucoplan 
Developed by UCB

Regimen B: Trial of Verdiperstat
Developed by Biohaven Pharmaceuticals

Regimen C: Trial of CNM-Au8
Developed by Clene Nanomedicine

Regimen D: Trial of Pridopidine
Developed by Prilenia Therapeutics

Regimen E: Trial of Trehalose (SLS-005)
Developed by Seelos 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

Full Trial Name: Neurodegenerative Alzheimer’s Disease and Amyotrophic Lateral Sclerosis (NADALS) Basket Proof of Concept Trial including Asymptomatic Individuals using Baricitinib

Trial Phase: Phase 1-2

Trial Length: Up to 28 weeks (Up to 7 in-person visits)

Drug to Placebo Ratio: No Placebo

Target: Type I interferon signaling

Science: Baricitinib aims to block type I interferon signaling, which is robustly active within the central nervous system of subsets of patients with Amyotrophic Lateral Sclerosis and Alzheimer’s Disease. Type 1 interferon signaling is an immune response that promotes inflammation which can lead to motor neurons dying and the progression of ALS symptoms. 

Administration: One 2 mg tablet once per day for the first 8 weeks of the trial, two 2 mg tablets once per day for the remaining 16 weeks. Tablets can be taken orally or crushed and administered through a G-tube

Purpose: In this study, the levels of baricitinib present in blood and cerebrospinal fluid (CSF) will be measured to determine safety and its effect on biomarkers related to ALS and AD. We hope these findings will help better evaluate the efficacy of baricitinib for the treatment of ALS. 

Principal Investigator: Doreen Ho, MD

Sponsor: Mark Albers, MD, PhD

Enrollment Contacts:

Sydney Hall, shall42@mgh.harvard.edu, 617-643-9005
Ali Wheeler, awheeler7@mgh.edu, 617-643-8449

Recruiting: People who have limited or no use of their hands, including people with ALS
Full Trial Name: BrainGate: 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

I'm interested!

Sponsor: Ionis Pharmaceuticals

Full Trial Name: A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients with Fused in Sarcoma Mutations (FUS-ALS)

Trial Phase: 1-3

Trial Length: Up to 3 years and 11 months (up to 20 in-person visits)

Participants: People with FUS ALS

Drug to Placebo Ratio: 2:1 for 14 months, open label extension (OLE) for 20 months

Target: FUS RNA

Science: ION363 is an investigational antisense medicine targeting the FUS gene to reduce production of the FUS protein. There is evidence that mutations in the FUS gene can lead to rapid, progressive loss of motor neurons in patients with FUS-ALS, so this drug may reduce or prevent disease progression in FUS-ALS patients.

Administration: Lumbar puncture (needle inserted into spinal fluid in the lower spine to administer dose)

Purpose: To evaluate the efficacy of the study drug in functioning and survival in ALS patients with FUS mutations.

Principal Investigator: Dr. Suma Babu

Sponsor: Ionis Pharmaceuticals

Enrollment contacts:
Alison Wheeler, awheeler7@mgh.harvard.edu, or 617-643-8449
Munaf Hatem, mhatem@mgh.harvard.edu, or 617-643-3530

Full Study Name: Cervical Collar Survey Study in ALS

Study Length: Single site visit

Participants: Diagnosis of ALS, or related motor neuron disease (MND)

Purpose: collect patient feedback on different cervical collars (neck braces) to help guide clinician recommendations around neck support.

Study Assessments: You will be asked to fill out several surveys at a single time point from your home. There are no in-person visits for this study. You can complete the surveys on your own time.

We will collect the following:
• Demographic and disease history information
• Information about neck pain and fatigue, and your use experiences with different neck braces
• Revised ALS Functional Rating Scale

Principal Investigator: Dr. Sabrina Paganoni

Sponsor: None

Enrollment Information: Please find the information sheet here

Study contact: If you have questions, please contact
Katey Burke, Physical Therapist, Katherine.Burke@mgh.harvard.edu, 617-726-1844

I'm interested!

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: James Berry, MD, MPH
Sponsor: ALS Finding a Cure, Target ALS, ALS Association, American Academy of Neurology/Muscular Dystrophy Association
Enrollment Contacts:
DIALS@mgh.harvard.edu, or call 
Courtney Uek, 617-724-0783
Rachel Freedman, 617-724-3268

DIALS Study Brochure

I'm interested! 

+Amyotrophic Lateral Sclerosis
+Asymptomatic ALS Gene Carriers
+Healthy Volunteers

Full Trial Name: A Longitudinal Analysis of Biomarkers in Patients with ALS 

Trial Length: 2 ½ years (7 in-person visits) 

Participants: People with ALS, asymptomatic ALS gene carriers, healthy volunteers 

Biomarkers: Blood, urine, and cerebrospinal fluid 

Purpose: To test potential biomarkers over time, which can be used to further uncover ALS pathophysiology, discover disease biomarkers, and identify new therapeutic targets. The biomarkers may help diagnose ALS sooner, monitor ALS progression, and teach us about potential causes and treatments for ALS. The samples we collect will be used to compare and analyze changes in immune cells and other changes in plasma and gene expression.

Principal Investigator: James Berry, MD, MPH 

Sponsor: Holy Cross Hospital, Inc.

Enrollment Contact: mgh_labpals_study@mgh.org 
Irene Chang, 617-724-7113
Annie Williams, 617-726-4284

I'm interested!

+Amyotrophic Lateral Sclerosis
+Asymptomatic ALS Gene Carriers
+Healthy Volunteers

Enroll and participate from your home!

Full Study Name: Longitudinal Assessment of the Gut Microbiome in People with ALS

Study Length: 5 years

Participants: People with ALS, asymptomatic ALS gene carriers, healthy volunteers

Biomarkers: Stool and blood samples

Purpose: To collect and analyze stool samples and observe the relationship between the gut microbiome and the progression of ALS over time. Information collected in this study will further our understanding of ALS and contribute towards the development of novel therapeutics

Principal Investigator: James Berry, MD, MPH
Sponsor: National Institutes of Health and Brigham and Women’s Hospital
Enrollment Contact: mgh-als-microbiome@mgb.org
Mia Fleischer, 617-724-5659
Megan Okoro, 617-643-6252

I'm interested!

Trial Length: 12 months, 5 in-person visits

Participants: People with ALS who walk with or without an assistive device 

Purpose of Study: We are using an app and two wearable sensor devices to create large data sets that clinicians can use to gain a better understanding of ALS progression. ALS researchers are exploring new ways to collect meaningful data to accelerate drug discovery and support the use of these technologies in clinical trials.

Study Assesments: The study lasts one year. Each participant will download an app and wear watch-like sensors on their wrist and ankle daily. A photo of the sensor on a wrist is included in this brochure. The app will prompt you to complete tasks every two weeks. We expect these tasks to take you about 5-10 minutes. Participants will also be asked to complete in person visits every three months. These visits can sometimes be done on the same day as other visits to Mass General.

Principal Investigator: James Berry, MD, MPH

Enrollment Contacts: ALSdigitalstudies@mgb.org

I'm interested!

Full Trial Name: Feasibility and Sensitivity of a Symptom Monitoring Application in Real Time (SMART) for ALS 

Trial Length: 12 months 

Participants: People with ALS and healthy volunteers 

Purpose of Study: To determine the usefulness of a smartphone app in collecting research data and to learn more about disease progression. 

Study Assessments: 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 study requires participants 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 Contact: alsdigitalstudies@mgb.org

I'm interested!

Enroll and participate from your home! 

Full Study Name: Social Determinants of Health and ALS Disease Progression

Study Length: Up to 5 remote survey sessions over 12 months 

Participants: People over the age of 18 with a diagnosis of ALS living in the US 

Purpose: In this research study, we want to learn about how non-medical factors known as Social Determinants of Health (SDOH) impact the progression of ALS. SDOH can include where a person lives,what career they chose, or whom a person lives with, The data collected from  this research can be used to develop targeted programs and resources that may improve the quality of life for families impacted by ALS.

Study Assessments: You will be asked to complete several surveys from your home every 3 months for up to one year. Each session will take 30-60 minutes. We may also collect relevant medical information from you throughout the study.

Principal Investigator: James D. Berry, MD

Enrollment Contacts: alsdigitalstudies@mgb.org

Sydney Hall, 617-643-9005
Sravan Mandepudi, 617-643-6036

I'm interested!

Study of Speech Motor Impairment in ALS

Full Study Name:  Speech motor impairments in ALS

Study Length: Up to 4 remote sessions, of up to 1.5 hours each

Participants: People with ALS

Purpose of Study: To learn more about speech symptoms experienced by people with ALS, in order to help improve the diagnosis and treatment of ALS

Study Assessments: 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

Principal Investigator: Dr. Jordan Green, Ph.D., CCC-SLP

Sponsor: National Institutes of Health

Enrollment Contacts: Speech and Feeding Disorders Lab staff, 617-724-6347, speechfeedinglab@mghihp.edu 

I'm interested!

Study of Target ALS - Coming soon!

Full Study Name:  Target ALS Biomarker Study: Longitudinal Biofluids, Clinical Measures, and At-Home Measures

Study Length: 16 months for ALS participants, 12 months for healthy volunteers

Participants: ALS patients and healthy volunteers able to have lumbar punctures

Biomarkers: Blood, spinal fluid, urine

Purpose of Study: The goal of the study is to build a library of samples (blood, cerebral spinal fluid, and urine) and linked medical and genetic data. Collaborating researchers will have access to this information to advance their knowledge of ALS.

Principal Investigator: Mark Garret, MD

Sponsor: Target ALS

Enrollment Contacts: targetals@mgb.org
Aisling Finnegan, 617-726-1880
Jingqi Zhu, 617-643-2522

I'm interested!

Full Trial Name: An open-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

Trial Length: 6 months

Drug to Placebo Ratio: Open label (no placebo)

Target: Inhibitor of CSF-1R

Science: In people with ALS, immune cells in the brain called microglia are activated and cause neuroinflammation. The goal of this study is to reduce neuroinflammation through treatment of BLZ945. This will be evaluated using PBR28 PET imaging to measure microglial activation, as well as through collection of biomarkers of neuroinflammation and disease activity in participants with ALS.

Administration: Oral pill taken once per week or for the first four days of a two-week period

Purpose: To learn about the safety and tolerability of BLZ945 in adults with a diagnosis of Amyotrophic Lateral Sclerosis (ALS) and to help select the most appropriate doses for the planning of future research in patients with ALS.

Principal Investigator: Suma Babu, MD, MPH

Sponsor: Novartis 

Enrollment Contacts:
Jingqi Zhu, jzhu34@mgh.harvard.edu, or 617-643-2522
Munaf Hatem, mhatem@mgh.harvard.edu, or 617-643-3530

Full Trial Name: A Phase 2a Trial to Evaluate the Safety, Tolerability, and Biological Activity of LAM-002A (apilimod dimesylate capsules) in C9ORF72-Associated ALS

Trial Phase: 2a

The aim of this Phase 2 research study is to find out if LAM-002A is a safe treatment option for patients with C9ORF72-associated ALS (C9ALS). During the study, participants will receive the LAM-002A drug orally in pill form. This is an open label extension trial, meaning that for the first 12 weeks, some participants will receive a placebo (which looks like the study drug, but has no active ingredients) instead of LAM-002A. Following the completion of weeks 1-12, all participants will be eligible to receive LAM-002A for the next 12 weeks of the trial. Participation in this study will involve blood draws, lumbar punctures, and several clinical measures of ALS, as research staff seek to understand how LAM-002A is processed by the body and if it impacts ALS progression. Our team is looking for patients with C9ALS and a slow vital capacity of ≥50% who are willing to complete 10 study visits and several phone calls over 28 weeks. Please reach out to the enrollment contact listed below: 

Principal Investigator: Suma Babu, MD, MPH
Sponsor: AI Therapeutics, Inc.
Enrollment Contact: Shea Golden, 617-724-3268 or sgolden3@mgh.harvard.edu
Allison Carey, 617-726-1559 or abcarey@mgh.harvard.edu

Full Trial Name: A Phase 2a Study of TPN-101 in Patients with C9ORF72 ALS/FTD (Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia) 

Trial Phase: 2 

Trial Length: 13-14 months (13 in-person visits) 

Participants: People with C9 ALS and/or FTD 

Drug to Placebo: 3:2 for 24 weeks, open label extension (OLE) for 24 weeks 

Target: Retrotransposon hL1 

Science: TPN-101 works to prevent hL1 expression, since hL1 may cause further ALS progression. HL1 is shown to be overexpressed in C9ALS causing DNA damage and neuroinflammation. TPN-101 may suppress hL1 expression to prevent progression. Ï

Administration: Gel capsule taken orally 

Purpose: To assess the safety and tolerability of the drug TPN-101 in patients with C9orf72 ALS and/or FTD. This study will also measure the levels and efficacy of the drug in your body over time. 

Principal Investigator: Dr. James D. Berry, MD, MPH 

Sponsor: Transposon Therapeutics, Inc. 

Enrollment Contact:
Jacqueline Topping, jtopping@mgh.harvard.edu , 617-643-6036

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:
Shea Golden, 617-724-3268, sgolden3@mgh.harvard.edu
Munaf Hatem, mhatem@mgh.harvard.edu, 617-643-3530

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
Trial Length: 6-7 months (13 in-person visits)
Drug to Placebo Ratio: 2:1 or 3:1, open label extension
Target: ATXN2 protein

Science:  BIIB105 is an antisense oligonucleotide (ASO) medication that may reduce the amount of ATXN2 protein. By decreasing ATXN2, this may prevent the accumulation of TDP-43 protein, which is responsible for the death of motor neurons.

Administration: Lumbar punctures (needle inserted into spinal fluid in the lower spine to administer dose)

Purpose: To learn about the safety and tolerability of BIIB105 in adults with a diagnosis of Amyotrophic Lateral Sclerosis (ALS) and to look at the level and action of the study drug in the body and what happens to this level over time.

Principal Investigator: . Suma Babu, MBBS, MPH
Sponsor: Biogen MA, Inc.

Enrollment Contacts:
Erica Scirocco, escirocco@mgh.harvard.edu, or 617-726-1363
Munaf Hatem, mhatem@mgh.harvard.edu, or 617-643-3530