Healey Center for ALS Passes 50% Participant Enrollment Milestone for First 3 Regimens in the HEALEY ALS Platform Trial

The HEALEY ALS Platform Trial, an initiative led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and conducted at sites of the Northeast ALS (NEALS) consortium, continues to gain momentum just months after launching in 2020, passing the 50% participant enrollment milestone for the first 3 regimens.

The platform trial approach speeds up scientific discovery, reduces trial start-up times, decreases costs, and allows data from participants in placebo groups to be shared. This results in high statistical power and allows more participants to receive active investigational products (rather than placebo).

As the first ever platform trial for amyotrophic lateral sclerosis (ALS), the HEALEY ALS Platform Trial is currently testing four investigational products and will add more over the next several months. The three experimental treatments with 50% enrollment include: Zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], developed by UCB; Verdiperstat, an oral myeloperoxidase inhibitor, developed by Biohaven Pharmaceuticals, and CNM-Au8 nanocrystalline gold, an intracellular nanocatalyst to support cellular bioenergetics developed by Clene Nanomedicine, Inc. A fourth investigational product— pridopidine, a highly selective Sigma-1 receptor (S1R) agonist developed by Prilenia — was added to the trial in early January of this year.

People with ALS are currently enrolled in the HEALEY ALS Platform Trial at several trial sites across the US. More NEALS consortium sites will be added in 2021 to expand trial access even further.

“We are very thankful that despite the challenges of COVID-19, all collaborators involved in the HEALEY ALS Platform Trial have come together to make sure people with ALS are safely able to enroll in this groundbreaking trial,” says Merit Cudkowicz, MD, director of the Sean M. Healey & AMG Center for ALS at Mass General and chief of the Department of Neurology.

“We are deeply grateful to trial participants and their families, and to the entire ALS patient community for their support: without their dedication and engagement, this trial would not be possible,” says Sabrina Paganoni, MD, PhD, investigator at the Sean M. Healey & AMG Center for ALS at Mass General “The sooner enrollment is complete, the sooner we will have answers about the safety and efficacy of these investigational products.”

The HEALEY ALS Platform Trial is perpetual and will continue to add more clinical sites, participants and investigational products until cures for everyone with ALS are discovered.

For more information, please visit the HEALEY ALS Platform Trial page.