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 - No Longer Enrolling 

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

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

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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

Sponsor: Rapa Therapeutics, LLC 

Full Trial Name: Phase 2/3 Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) for Amyotrophic Lateral Sclerosis 

Trial Phase: 2/3

Trial Length: Up to one year in person (5-8 visits). Two years of remote follow-up (8 visits)

Drug to Placebo: Open Label (no placebo)

Target: T-cells 

Science: In people with ALS, the body’s immune system becomes imbalanced, which is thought to contribute to the loss of motor neurons in the brain and spinal cord. Regulatory T-cells, a specific type of immune cell, reduce inflammation. Therefore, scientists believe that they could help to balance the immune system of people with ALS. The goal of this study is to utilize a modified version of Regulatory T-cells, called RAPA-501 cells, to reduce neuroinflammation and potentially slow ALS progression. This process involves: (1) harvesting T-cells from the participants own blood through a process called apheresis, (2) reprogramming the harvested T-cells in special cell culture conditions to become RAPA-501 cells, and (3) infusing the specialized RAPA-501 cells back into the participants bloodstream through an IV.

Administration: 

(1) Apheresis (blood separation) to collect T-cells 

(2) Intravenous (IV) infusion of the specialized RAPA-501 cells 

Purpose: To learn more about the efficacy and safety of RAPA-501 cell therapy in people living with ALS

Principal Investigator: James Berry, MD, MPH

Enrollment Contacts:
Megan Okoro, 617-643-6252, mokoro@mgh.harvard.edu
Kayla Furney, 617-643-7828, kfurney@mgh.harvard.edu

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 Study Name: ASSESS ALL ALS – Longitudinal Biomarker Study for Symptomatic ALS and Healthy Control Participants  

Study Length: up to 2 years (7 inperson or remote visits) 

Participants: People with ALS and healthy volunteers 

Biomarkers: Blood 

Purpose: To study people diagnosed with ALS and healthy participants to further our understanding of the disease and potential biomarkers of disease progression. The information collected in this study may contribute to future research and development of new treatments for ALS and similar neurological diseases. 

Principal Investigator: James Berry, MD, MPH  

Sponsor: National Institutes of Health and St. Joseph’s Hospital and Medical Center, Phoenix, AZ 

Enrollment Contacts: mghassessallals@mgb.org
Aisling Finnegan, 617-726-1880
Miranda Durcan, 617-643-9550

Read ASSESS ALL ALS 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
Carolyn Dwyer, 617-724-7928

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
Carolyn Dwyer, 617-724-7928
Mia Fleischer, 617-724-5659

I'm interested!

Full Study Name: PREVENT ALL ALS – Longitudinal Biomarker Study for Participants Who are At Risk for ALS

Study Length: up to 3 years (6 remote visits/3 yearly in-person visits)

Participants: People who are asymptomatic ALS gene carriers or have a family history of ALS

Biomarkers: Blood and optional cerebrospinal fluid collection

Purpose: To study people at risk for developing ALS and broaden our understanding of causes of underlying early disease changes. The information collected in this study may result in the development of treatments that target the earliest changes in ALS and lead to possible disease prevention.

Principal Investigator: James Berry, MD, MPH

Sponsor: National Institutes of Health and St. Joseph’s Hospital and Medical Center, Phoenix, AZ

Enrollment Contacts: mghpreventallals@mgb.org
Courtney Uek at 617-724-0783
Rachel Freedman at 617-724-3268

Read PREVENT Brochure

I'm interested! 

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!

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: James Berry, MD, MPH

Sponsor: Target ALS

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

I'm interested!