To present the design and launch of the first platform trial for ALS. 

There is an urgent need for new ALS therapies. Given the large number of investigational products being developed, we designed an adaptive platform trial to evaluate multiple experimental agents simultaneously under a single master protocol and increase the efficiency of ALS drug development.

The HEALEY ALS Platform Trial is a perpetual, multi-center, randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of multiple investigational products, with sharing of placebo participants across treatments. The trial is designed to provide supportive as well as potentially confirmatory evidence for therapies found to slow disease progression. The design and launch of this trial is the result of an active collaboration among international ALS clinical trial experts and statisticians, with input from the patient community, industry, and regulatory authorities. 

The trial received a “May Proceed” notice from the FDA for the first three regimens in January 2020 and is scheduled to start enrolling participants at 54 NEALS consortium sites in early 2020. Participants will be randomized in two stages; first to a treatment regimen testing a specific experimental agent, followed by 3:1 randomization to either active or placebo within a regimen. Participants are followed for 24 weeks, and regimens are strongly encouraged to offer open-label extensions. The sample size for each regimen is 160 (120 active treatment, 40 placebo), with sharing of placebo participants across regimens. Compared to traditional drug development, we estimate that the trial will find an effective therapy three-fold faster with two-thirds as many participants and one-third as many on placebo.

The HEALEY ALS Platform Trial is being launched with the first three regimens.  More are scheduled to be added in late 2020. The trial is poised to accelerate the development of new treatments for ALS.