Sean M. Healey & AMG Center for ALS Announces Integration of Placebo Data from Regimens A through E of the HEALEY ALS Platform Trial into PRO-ACT Database

The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) is pleased to announce the successful integration of placebo data from the first five regimens from the HEALEY ALS Platform Trial into the PRO-ACT database. This vital resource is now available to researchers working to advance our understanding of ALS and discover new treatments. We are committed to data sharing to accelerate ALS research.

The HEALEY ALS Platform Trial is designed to enhance the feasibility, efficiency, and impact of ALS trials. Recently, we made important updates to the trial's Master Protocol to increase the likelihood of detecting benefits from investigational products and accelerating discovery of new treatments for ALS. These changes include extending the duration of the Randomized Controlled Trial (RCT) from 24 to 36 weeks, allowing more comprehensive data collection and a longer evaluation period for treatments. The inclusion criteria were adjusted, with the time since symptom onset now set at 24 months, down from the previous 36-month threshold. The visit schedule was made more flexible, offering increased opportunities for remote visits in the Active Treatment Extension (ATE), and novel biomarkers were included.

With these updates, placebo data from the first five regimens (A to E) have been “retired” and won’t be used in the analysis of future regimens. However, this rich dataset is now part of the PRO-ACT database, ensuring its ongoing contribution to future research and to those affected by ALS. Importantly, this amplifies the impact of individual trial participants, as their contributions to earlier regimens continue to advance scientific understanding and drive progress toward effective treatments well beyond the original scope of the trial. Our updated Data & Report Sharing Policy promotes the responsible sharing of these valuable data and associated participant samples - fostering collaboration, accelerating scientific discovery, and helping move the field forward for everyone affected by ALS.

“We are thrilled to announce this integration, which provides researchers with important data that can lead to new discoveries in ALS research,” said Merit Cudkowicz, M.D., principal investigator and sponsor of the HEALEY ALS Platform Trial, director of the Sean M. Healey & AMG Center for ALS, and the inaugural executive director of the Neuroscience Institute at Mass General Brigham. “Open science and collaboration are essential to driving progress in ALS. By making this information available through the PRO-ACT database, we hope to foster research partnerships and accelerate the development of effective therapies for people living with ALS.”

“The PRO-ACT database has already had a transformative impact on ALS research - helping to design smarter clinical trials, uncover new prognostic factors, and develop novel predictive models. It’s incredibly encouraging to see the resource continue to grow, providing even more opportunities for discovery and accelerating progress for the ALS community,” said Alex Sherman, Director of the Center for Innovation and Biomedical Informatics (CIB) at the Neurological Clinical Research Institute (NCRI) and Principal Associate in Neurology at Harvard Medical School. “We extend our heartfelt thanks to everyone who made PRO-ACT possible - the companies, the researchers, the clinicians, and most of all, the ALS patients past and present around the world, to whom this initiative is dedicated.”

The Sean M. Healey & AMG Center for ALS is committed to improving the lives of those affected by ALS through research, education, innovation and collaboration. This milestone marks a significant advancement in our efforts to understand this complex disease and develop new therapeutic options.

Researchers and interested parties are encouraged to explore the PRO-ACT database and leverage the newly available data to contribute to the ongoing fight against ALS.

De-identified placebo data from the first five regimens (A to E) may also be requested directly from the Healey & AMG Center here.

For further information about the HEALEY ALS Platform Trial please visit: HEALEY ALS Platform Trial

About the PRO-ACT Database

The PRO-ACT (Pooled Resource Open-Access ALS Clinical Trials) database is the world’s largest open collection of clinical trial data in ALS. Created to accelerate research and drug development, PRO-ACT brings together data from thousands of people who have participated in past ALS clinical trials.

By combining and sharing this information in one central place, researchers around the globe can uncover new patterns, identify promising treatment targets, and improve how future ALS trials are designed. The database is freely available to the research community and has already led to important scientific discoveries that bring us closer to better therapies for ALS.

For further information about the PRO-ACT Database please visit: PRO-ACT Database

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 many 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.