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 Community Q&A Webinars to stay connected to trial news and updates.  

Current Active Regimens - No Longer Enrolling 

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

Regimen F: Trial of ABBV-CLS-762
Developed by Calico in collaboration with AbbVie

Regimen G: Trial of DNL343

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

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
Shannon Chan, 617-643-4968, schan33@mgh.harvard.edu

Sponsor: Amylyx Pharmaceuticals

Full Trial Name: A Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of the Antisense Oligonucleotide AMX0114 Administered to Adult Participants with Amyotrophic Lateral Sclerosis

Trial Phase: 1

Trial Length: 25 weeks

Participants: People 18 years or older with ALS

Drug to Placebo Ratio: 3:1

Target: CAPN2 RNA

Science: AMX0114 is an investigational antisense oligonucleotide (ASO) medicine targeting the CAPN2 gene to reduce production of the Calpain-2 protein. There is evidence that calpain-2 is associated with processes known to cause neuronal injury and loss of axons which are attached to the motor neurons. By reducing the Calpain-2 protein this drug may slow disease progression.

Administration: Lumbar puncture (needle inserted into spinal fluid in the lower spine to administer dose); 4 doses every 4 weeks with an additional lumbar puncture for collection at the end of the study

Purpose: To evaluate the safety and tolerability of the study drug in ALS patients

Principal Investigator: Dr. Sabrina Paganoni

Enrollment Contacts: amx0114healey@mgb.org
Vanessa Travero, 617-724-2069
Lucy Lee, 617-643-7434

Sponsor: Coya Therapeutics

Full Trial Name: Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multi-Center, 24-Week Study with Additional 24-Week Blinded Active Extension to Evaluate the Safety and Efficacy of COYA 302 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Trial Phase: 2

Trial Length: 24-week placebo-controlled trial followed by 24-week extension

Participants: Adult participants (≥18 to ≤75 years of age) with sporadic or familial ALS

Drug to Placebo Ratio: 2:1

Target: Increase Regulatory T Cells (Tregs)

Science: Tregs which reduce inflammation are often present in fewer numbers in people with ALS. COYA 302 is a combination of interleukin-2 (IL-2) and a biosimilar candidate to abatacept. This investigational drug is expected to increase the function and number of Tregs while also directly reducing inflammation. Improving the efficacy of Tregs may reduce toxicity to the motor neurons and slow ALS progression.

Administration: Subcutaneous injection

Purpose: To study the safety and efficacy of COYA302 in participants with ALS.

Principal Investigator: James Berry, MD, MPH

Enrollment Contacts: coya302healey@mgb.org
Jahnavi Murthy, 617-726-3015
Sophie Cohen, 617-643-7828

Sponsor: Zydus Therapeutics

Full Trial Name: A phase 2b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Usnoflast administered to adult subjects with Amyotrophic Lateral Sclerosis (ALS)

Trial Phase: 2

Purpose of Trial: To evaluate the efficacy and safety of Usnoflast in participants with ALS.

Trial Length: 36 weeks

Participants: Adults with ALS

Drug to Placebo Ratio: 1:1:1 (Usnoflast: Usnoflast+Placebo: Placebo)

Target: NLRP3 inflammasome

Science: Usnoflast is a selective inhibitor that prevents the activation of the NLRP3 inflammasome pathway. This pathway is believed to contribute to ALS disease progression through activating proteins that lead to neuroinflammation and cell death.

Administration: Participants will receive an oral dose of the study drug twice daily as a tablet. Participants will be asked to fast two hours before and after each dose.

Lumbar Punctures: 3 optional

Principal Investigator: Jennifer Morganroth, MD, MBA

Enrollment Contacts: usnoflasthealey@mgb.org
Kimberly Lin, 617-724-3268

Sponsor: VectorY Therapeutics B.V.

Full Trial Name: A Phase 1/2 Study of the Safety and Tolerability of ICM VTx-002 in participants with ALS (PIONEER-ALS)

Trial Phase: 1/2

Trial Length: Approximately 5 years. Year 1: 12 in-person visits and 4 remote visits. Years 2-5: 8 in-person (preferable) or remote visits

Participants: People diagnosed with ALS without SOD1or FUS mutations

Drug to Placebo Ratio: Open label, no placebo

Target: TDP-43 protein clumps

Science: Participants will receive a single injection into the intra-cisterna magna (at the base of the brain) of the disease-modifying gene therapy VTx-002. The goal of VTx-002 is to target clumps of TDP-43 protein, which are believed to contribute to cellular dysfunction and neuronal death. VTx-002 is delivered by a virus vector (harmless virus turned into a delivery tool that carries information into the cells). This study drug aims to break down TDP-43 clumps and restore their function to prevent disease progression.

Administration: Intra-cisterna magna injection (at the base of the brain) performed by neurosurgeon

Purpose: To assess the safety and tolerability of VTx-002 for the treatment of people with ALS

Principal Investigator: Doreen Ho, MD

Enrollment Contacts:
Megan Okoro, pioneeralshealey@mgb.org, 617-643-6252
Vanessa Travero, pioneeralshealey@mgb.org, 617-724-2069

Sponsor: ProJenX, Inc.

Full Trial Name: A Phase 1, Randomized, Double-Blind, Placebo-Controlled Study with ProJenX to Evaluate Safety, Tolerability, and Pharmacokinetics of Prosetin with an Optional Open-Label Extension

Trial Phase: 1

Trial Length: Approximately 28 days for Blinded Treatment; Approximately 23 months for Open-Label Extended Treatment with remote visits after 6 months

Participants: Adults with ALS

Drug to Placebo Ratio: 3 drug to 1 placebo

Target: MAP4 Kinase

Science: This drug is a molecule that inhibits a protein known as the MAP4K pathway. This is thought to have neuro protective properties and slow inflammation.

Administration: Participants will receive an oral dose of the study drug once daily. The dose will be administered through a syringe. Participants will be asked to fast two hours before and one hour after each dose.

Lumbar Punctures: 5 total – 1 during the placebo-controlled portion, 4 during the open-label extension

Purpose: To determine the safety and pharmacokinetics of Prosetin

Principal Investigator: James Berry, MD, MPH

Enrollment Contacts:
Shyanne Hill, 617-643-5376, sthill@mgh.harvard.edu
Kimberly Lin, 617-724-3268, klin17@mgh.harvard.edu

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 Contact: 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 
Lada Filippov, 617-724-7048
Carolyn Dwyer, 617-724-7928

+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
Lada Filippov, 617-724-7048

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
Anika Allen, 617-724-9196

Read PREVENT Brochure

I'm interested! 

Full Study Name: Respiratory Comorbidity Detection Using Digital Devices (Empatica)

Sponsor: ALS Association

Study Length: 18 Months

Participants: People living with ALS who qualify for breathing support and use assistive devices for mobility. We are also looking for volunteers who have not been diagnosed with ALS or have a relative with confirmed genetic ALS.

Purpose of Study: We aim to improve monitoring of respiratory complications like pneumonia, pulmonary embolisms or deep vein thromboses in people living with ALS using smartwatch sensors (Empatica device). The study could help develop better detection and treatment methods for these respiratory complications related to ALS.

Study Assessments: Participants will have visits every three months for a total of seven in person visits. At the visits, participants will undergo imaging, blood tests, ALS clinical exams, and symptom surveys.

Principal Investigator: James Berry MD, MPH

Enrollment Contacts: Sravan Mandepudi, 617-643-6036 or ALSdigitalstudies@mgb.org

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: Jordan Green, PhD, 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: Speech, Social Connectedness, and Well-being Outcomes in ALS

Study Length: 7 months

Participants: People with ALS

Purpose of Study: To understand the impact of ALS and associated speech impairments on social connectedness and quality of life.

Study Assessments: The study asks each participant to use a smartphone for a few minutes a day and to complete surveys, and record your voice at two time points about 6 months apart. You will also meet with a researcher over video two times to complete surveys; and mobility, cognitive, and speech tasks. The smartphone will collect passive data for the first and last month of the study.

Principal Investigator: Kathryn Connaghan, PhD

Enrollment Contact: ALSdigitalstudies@mgb.org, or Mackenzie DeMello, 617-643-2499

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

I'm interested!

Full Trial Name: Electrical Impedance Myography via the Myolex mScan as an ALS Biomarker

Trial Length: 8-10 months

Participants: Diagnosed with ALS, 18+ years of age, symptom onset ≤ 36 months, Vital Capacity ≥ 50% of predicted capacity as measured by forced vital capacity, can identify a study partner for home visits

Purpose of Study: This study is being done to see how a device, the mScan, measures muscle changes over time in people living with ALS. This device uses Electrical Impedence Myography (EIM), which uses a very small, non-invasive (e.g. no needles), brief (about 6 seconds), and painless electrical current to measure the muscle tissue. We hope that this measurement can help with tracking disease progression and be used in the future to help understand how well treatments are working.

Study Assessments: EIM measurements will be taken with the mScan device at your regular clinic visits, at least 3 times and up to 5 times in an 8-10 month period. Your study partner will also take mScan measurements twice a week at home. The ALS Functional Rating Scale-Revised (ALSFRS-R) will be completed weekly at home and then also at the in-person visits.

Principal Investigator: Sabrina Paganoni, MD, PhD

Enrollment Contacts: Lindy Feintuch, lfeintuch@mgh.harvard.edu, 617-724-0783, or email the team at alsdigitalstudies@mgb.org