Jeremy Shefner, MD, PhD and a team of researchers from the HEALEY ALS Platform Trial Biomarkers and Outcomes Measures taskforce have released a poster at the European Network to Cure ALS 2022 International Meeting that includes data around baseline speech assessment and vital capacity (VC), a measure of respiratory function, which were measured remotely as part of the HEALEY ALS Platform Trial.

The HEALEY ALS Platform Trial is an innovative effort to streamline ALS clinical trials by reducing placebo treatment assignment and maintaining an active network of sites studying continually enrolling participants into treatment regimens. Participation includes 24 weeks of placebo-controlled treatment. The first 4 regimens have completed enrollment. Standard ALS outcomes are assessed as well as exploratory outcomes, including coached home spirometry (Zephy Rxapp) and quantitative voice characterization (Aural Analytics) using a mobile app.

Eight hundred and seventy participants with ALS were consented and screened; 653 were randomized within the first 4 regimens.  Results show that VC can be reliably measured at home under supervision and that sustained phonation is a good predictor of VC when a spirometer is unavailable.

“This study showed that the HEALEY ALS Platform Trial will generate a wealth of data on new outcome measures in ALS and that novel respiratory and speech related outcome measures will produce important data relevant to the design of future trials,” said Merit Cudkowicz, MD, MSc, director of the Healey Center for ALS. “This is a big step in our mission to find new treatments and cures for ALS.”