Sean M. Healey & AMG Center Announces Updates to HEALEY ALS Platform Trial Master Protocol

BOSTON- In 2020, investigators at the Healey & AMG Center for ALS launched the HEALEY ALS Platform Trial, the first of its kind clinical trial for Amyotrophic Lateral Sclerosis (ALS). The trial is a multicenter, double-blind, placebo-controlled, perpetual and adaptive study evaluating the safety and efficacy of multiple investigational products for the treatment of ALS. Today, the Healey & AMG Center announced updates to the Master Protocol for the trial. 

The platform trial’s perpetual and adaptive design allows for the integration of new information based on experience, the analysis of data, and feedback from participants. The data collected in the initial regimens tested in the platform trial has been meticulously studied by leaders in the field including investigators, biostatisticians, and industry partners. With this information, the team has made the following updates to the master protocol to facilitate future learning in the trial. 

The duration of the Randomized Controlled Trial (RCT) period is extended from 24 to 36 weeks to allow for a longer treatment evaluation period and increase the power to see positive results from treatments that might take longer to have an effect. This extension will enable researchers to more effectively assess the long-term benefits of treatments that may require additional time to show their full potential. By allowing for a more comprehensive evaluation, the extended trial duration enhances the ability to detect meaningful changes in patient outcomes, particularly for therapies with slower onset of action, ultimately providing a clearer picture of their efficacy in treating ALS. Additionally, the inclusion criteria were modified, with the time since symptom onset now set at 24 months.

We have continued to refine the patient-centric aspects of the trial: the visit schedule has been made more streamlined and flexible, offering increased opportunities for remote visits in the Active Treatment Extension (ATE). Another notable update is the addition of peripheral blood mononuclear cell (PBMC) collection, which will be banked for future generation of induced pluripotent stem cells (iPSCs), providing valuable insights for research and therapy development.  

These changes were presented by Lori Chibnik, PhD at a plenary session at the International MND Symposium in Montreal, Canada on December 7, 2024.

In addition to the team of investigators, biostatisticians, and industry partners, changes to the master protocol have been shaped by the invaluable feedback from the HEALEY ALS Platform Trial Patient Advisory Committee. This committee, made up of individuals living with ALS and their caregivers, has played a crucial role in guiding the trial’s design to ensure it aligns with the needs and experiences of the ALS community. 

“We learned a lot from the first regimens in the HEALEY ALS Platform Trial,” said Merit Cudkowicz, M.D., M.Sc., principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, chair of the Department of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “Building on the insights we've gained, these updates to the master protocol will significantly strengthen the trial’s ability to identify effective treatments for ALS and bring us closer to finding answers for this devastating disease.”  

Results of the first five regimens have previously been shared with two of the five planning for follow up phase 3 testing (regimens C and D). Regimen F and G have completed enrollment, and results will be shared when available. The team at the Healey & AMG Center is working with several new companies to launch new regimens in 2025. 

For more information about the HEALEY ALS Platform trial, join our Community Q&A Webinar Series on select Thursdays at 5pm EST. 

To learn more about collaborating with the Healey & AMG Center on the HEALEY ALS Platform Trial, click here 

To learn more about participating in the trial, contact the Patient Navigation Team 

About the Sean M. Healey & AMG Center for ALS at Mass General  

At the Sean M. Healey & AMG Center for ALS at Mass General, we are committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development.  

Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it.  

With dozens of active clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care. Together, we will find the cures.