The HEALEY ALS platform trial, which is evaluating the safety and efficacy of several potential treatments for amyotrophic lateral sclerosis (ALS), has passed the 50% participant enrollment target for its first three regimens.
The investigational treatments are UCB’s zilucoplan, Biohaven Pharmaceuticals’ verdiperstat, and Clene Nanomedicine’s CNM-Au8. A fourth candidate — Prilenia’s pridopidine — was added to the trial this January, and more are planned to be included.
“We are excited to achieve this enrollment milestone,” Robert Glanzman, MD, chief medical officer of Clene, said in a press release.
HEALEY is led by ALS specialists of the Sean M. Healey & AMG Center for ALS at Mass General Hospital in collaboration with the Northeast ALS consortium clinical trial network.
“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,” Merit Cudkowicz, MD, director of the center, said in another press release.
As a platform trial, HEALEY is evaluating the potential of multiple ALS treatment candidates simultaneously, with a goal of speeding the development of those that seem to be the most promising.
A total of 160 participants will be included in each of the trial’s regimens. All will be randomly assigned to receive either the investigative treatment or a placebo twice daily for 24 weeks. For every three patients on active treatment, one will be given a placebo.
The trial’s main goal is to assess patients’ changes in ALS severity over time, as measured by the ALS Functional Rating Scale-Revised. Secondary objectives include changes in respiratory function, muscle strength, and survival.
By studying multiple investigational therapies in parallel, HEALEY was designed to maximize resources and speed the development of candidates that appear most promising. The approach also decreases costs, improves comparisons of several therapy candidates, and leads to stronger data by combining results from placebo patients across all groups.
“We are pleased that enrollment in the trial is progressing rapidly and very grateful to our collaborators at the Healey Center and the ALS research community for their leadership, boundless enthusiasm, and perseverance,” Irfan Qureshi, MD, verdiperstat development lead and vice president at Biohaven, said in a company press release.
“We are also deeply thankful to people with ALS and their families for their commitment to the HEALEY ALS Platform Trial,” added Qureshi.
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