CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial.

ABSTRACT:

Importance

Bioenergetic failure has been proposed as a driver of amyotrophic lateral sclerosis (ALS). CNM-Au8 is a suspension of gold nanocrystals that catalyzes the conversion of nicotinamide adenine dinucleotide hydride into NAD+, resulting in an increase of cellular adenosine triphosphate production.

Objective

To determine the effects of CNM-Au8 on ALS disease progression.

Design, setting, and participants

CNM-Au8 was tested as a regimen of the HEALEY ALS Platform Trial, a phase 2/3, multicenter, randomized, double-blind platform trial. The study was conducted at 54 sites in the US from July 2020 to March 2022 (final follow-up, March 17, 2022). A total of 161 participants with ALS were randomized to receive CNM-Au8 (n = 120) or regimen-specific placebo (n = 41). Da

SUBMITTER: Writing Committee for the HEALEY ALS Platform Trial 

PROVIDER: S-EPMC11833661 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature