HEALEY ALS Platform Trial

Overview

The HEALEY ALS Platform Trial, modeled after successful platform trials in oncology, is the first trial of its kind for people living with amyotrophic lateral sclerosis (ALS). In disorders like ALS, where there is a robust pipeline of therapeutics, platform trials offer the most efficient approach to accelerate therapy development. The goal is to test several new investigational products in separate regimens using the same trial infrastructure. With the ability to efficiently add and drop regimens and share control group data across regimens, platform trials provide more opportunities to find groundbreaking therapies at a faster pace, for less cost and with fewer participants on placebo than traditional clinical trials.

Within the first five years of its launch, the HEALEY ALS Platform Trial tested seven investigational products in the time that traditional clinical trials may have only tested one or two. Powered by a collaboration with the Network of Excellence for ALS (NEALS), the trial operates across an expanding consortium of research centers nationwide. The HEALEY ALS Platform Trial study team continues to evaluate and add new investigational products for inclusion in the trial with support from multiple stakeholders.

The perpetual and adaptive design of the Platform Trial allows for the integration of new information based on experience, the analysis of data, and feedback from participants. The Platform Trial now enrolls under the Next Generation Design, a refined master protocol that incorporates learnings from the first several years of the study. The Platform Trial is expected to remain active until safe and effective treatments are found for all people living with ALS.

Patients: For information about participating in the trial, please email HEALEYALSPlatform@mgh.harvard.edu or call 833-425-8257 (HALT ALS).

Researchers and industry: To collaborate with us on new regimens or trial sites, please email HealeyCenterforALS@mgh.harvard.edu.

Past Regimens with Active Clinical Development Programs in ALS

Data collected in two of the past regimens in the HEALEY ALS Platform Trial allowed the investigational drugs to move forward to the next phase of testing:

• Regimen C: CNM-Au8 by Clene Nanomedicine, Inc.
• Regimen D: Pridopidine, by Prilenia Therapeutics

Trial Sites

The HEALEY ALS Platform Trial is powered by a nationwide collaboration between research centers in the Network of Excellence for ALS (NEALS) and the Sean M. Healey & AMG Center. The trial is active at several sites across the U.S., making research more accessible. See below for a list of participating sites and their contact details, or visit ClinicalTrials.gov.

If you would like assistance connecting with a local site, please email HEALEYALSPlatform@mgh.harvard.edu or call 833-425-8257 (HALT ALS).

HEALEY ALS Platform Trial Indirect Cost Policy

In executing the Master Clinical Trial Agreement (MCTA) to participate in the HEALEY ALS Platform Trial, Sites agree they will be paid indirect costs (IDC) at the rate at which the originating funding organization reimburses MGH. For payments made from internal MGH philanthropic funds, the IDC will be capped at 15% of total direct costs.

For Patients

The Healey & AMG Center hosts public Q&A webinars for the ALS community. Join us at 5:00-5:30 pm Eastern Time on select Thursdays to discuss news and updates about the HEALEY ALS Platform Trial, ask questions, and hear from doctors and industry professionals at Mass General Brigham and beyond. Register to attend upcoming community Q&A webinars

If you have questions about the HEALEY ALS Platform Trial, please contact the Patient Navigator by email at HEALEYALSPlatform@mgh.harvard.edu or by phone at 833-425-8257 (HALT ALS).

Participant Resources

• Platform Trial Brochure
• To learn more about lumbar punctures, please visit the Clinical Trials and Studies webpage

Find Out If You're Eligible

We've prepared a short list of questions to help you find out if you might be eligible to participate in the HEALEY ALS Platform Trial. Please note that the result of this survey does not serve as official confirmation of your eligibility, as many factors are considered prior to study participation (view a full list of inclusion / exclusion criteria here). Determining eligibility for the Platform Trial will depend on a thorough assessment of your clinical symptoms, review of past medical history, and lab work that can only be performed by an investigator (doctor or nurse practitioner) on the trial.

Investigator Resources

Apply to Join the Platform Trial

• Requests for proposals
• Application form
• Confidentiality agreement template

Master Protocol

• HEALEY ALS Platform Trial master protocol
• REGIMEN-SPECIFIC APPENDIX

Policies

• Biosample Sharing Policy
• Data and Report Sharing Policy
• Data and Biosample Request Form
• Publication Policy
• Publication Form

Publications and Past Regimen Results

• Pridopidine treatment in ALS: subgroup analyses from the HEALEY ALS Platform trial [December 2025]
• Financing drug development via adaptive platform trials [July 2025]
• Operational Development and Launch of an Adaptive Platform Trial in Amyotrophic Lateral Sclerosis: Processes and Learnings from the First Four Regimens of the HEALEY ALS Platform Trial [May 2025]
• Safety and Efficacy of Trehalose in Amyotrophic Lateral Sclerosis (HEALEY ALS Platform Trial): An Adaptive, Phase 2/3, Double-blind, Randomised, Placebo-controlled Trial [May 2025]
• Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial [February 2025]
• Verdiperstat in Amyotrophic Lateral Sclerosis: Results from the Randomized HEALEY ALS Platform Trial [February 2025]
• CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial [February 2025]
• Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial [February 2025]
• Lessons from the HEALEY Adaptive Platform Trial in Amyotrophic Lateral Sclerosis [November 2024]
• Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis [May 2023]
• Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development [December 2021]