The Sean M. Healey & AMG Center for ALS Announces Completion of Enrollment for Regimen I in the HEALEY ALS Platform Trial Evaluating NUZ-001

• Enrollment is complete for Regimen I of the HEALEY ALS Platform Trial
• 250 participants were enrolled in less than 5 months, making this the fastest regimen to activate sites and enroll in the HEALEY ALS Platform Trial
• Thank you to people living with ALS and their families for participating in the rapid enrollment of this regimen, allowing for faster therapy development

The Sean M. Healey & AMG Center for ALS at Mass General Brigham is pleased to announce the completion of enrollment for Regimen I of the HEALEY ALS Platform Trial (ClinicalTrials.gov ID: NCT07410806). Regimen I evaluates the investigational product NUZ-001 developed by Neurizon Therapeutics. This is the first regimen to complete enrollment using the updated Next Generation Platform Trial master protocol.

The HEALEY ALS Platform Trial is a multicenter, double-blind, placebo-controlled trial for amyotrophic lateral sclerosis (ALS) created in partnership with the Network of Excellence for ALS (NEALS). The Platform Trial’s perpetual and adaptive design allows researchers to update and improve the master protocol as new information is gathered. Regimen I is the first trial to complete enrollment under the Next Generation master protocol, a refined protocol that incorporates learnings from the first several years of the Platform Trial. The revised protocol increases statistical power of the trial, streamlines operations, and adds more patient-centered features.

“We are excited to have met this important milestone for Regimen I,” says Merit Cudkowicz, MD, MSc, Director of the Healey & AMG Center, Executive Director of the Mass General Brigham Neuroscience Institute, and the Carol and James Herscot Endowed Neuroscience Institute Chair – Executive Director. “We had a record enrollment of 250 participants enroll in less than five months, which is a reflection of the dedication of the ALS community and high quality site staff at our NEALS sites.”

In May of 2026, the HEALEY ALS Platform Trial team expanded the regimen sample size from 160 to 240 participants because of faster than expected enrollment. With Regimen I serving as the only regimen currently enrolling in the Platform Trial, this change preserves the 3 to 1 randomization of the trial and the statistical power.

“We are thrilled to reach enrollment completion faster than any previous regimen in the Platform Trial, even with expanding the sample size” says Sabrina Paganoni, MD, PhD, Co-Principal Investigator of the HEALEY ALS Platform Trial and Co-Director of the Neurological Clinical Research Institute (NCRI) at Mass General Brigham. “After learning from the past seven regimens, we continued to make refinements to the already strong trial infrastructure, which led to a significant acceleration in start-up times and increasing enrollment speed for this regimen.”

Regimen I evaluates NUZ-001, an oral therapy that targets key pathological mechanisms in ALS, including TDP-43 protein aggregation and impaired autophagy, which are central features across multiple neurodegenerative diseases. Initial Phase 1 studies support favorable safety and pharmacokinetics of the drug. NUZ-001 is developed by Neurizon Therapeutics Limited, a biotechnology company based in Melbourne, Australia, and is not yet approved for use in any country.

Regimen I is co-led by Michael Weiss, MD, Director of the Neuromuscular Diseases Division at the University of Washington, and James Berry, MD, MPH, Director of the Mass General Brigham Neuroscience Institute Clinical Trials Center for Neurotherapeutics, Chief of the Division of ALS and Motor Neuron Diseases, and Director of the NCRI.

“It is inspiring to see sites activate and enroll quickly in such a short amount time,” says Dr. Weiss. “In platform trials, with each new regimen we learn from the previous one and adjust to activate sites faster and enroll more efficiently, which allows us to get results faster. We strongly believe in the “ALS Clock” and need for speed and efficiency. We are grateful for the enthusiasm from the ALS community for this regimen and helping us get to answers sooner.”

“We look forward to continuing the operation of this trial and learning more about the effects of NUZ-001 in people living with ALS,” says Dr. Berry. “Thank you to the participants and their families, sites, industry partners, and collaborators on this regimen. This progress would not be possible without your dedication and hard work.”

For further information about the HEALEY ALS Platform Trial, please visit our website.

About the HEALEY ALS Platform Trial

The HEALEY ALS Platform Trial is a multicenter, double-blind, placebo-controlled, adaptive trial for amyotrophic lateral sclerosis (ALS) created in partnership with the Network of Excellence for ALS (NEALS). The goal of the HEALEY ALS Platform trial is to accelerate the development of potential new ALS therapies. The trial tests and evaluates multiple investigational drugs simultaneously, shares infrastructure across trial sites, and improves start up and enrollment efficiencies, allowing for fast results.

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

At the Sean M. Healey & AMG Center for ALS at Mass General Brigham, 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.

About the Neurological Clinical Research Institute

The Neurological Clinical Research Institute (NCRI) at Mass General Brigham is an academic research organization composed of innovative researchers experienced and passionate about designing, developing, facilitating, and conducting multicenter clinical trials in neurological diseases. Our mission is to accelerate translational research in neurological disorders by initiating clinical development of novel therapies and leading trials of these compounds. We strive to be at the cutting edge of innovative trial design to accelerate therapy development by creating new trial methodology, discovering novel biomarkers and refining outcome measures.