HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose

Phase 2/3 Completed 161 enrolled Merit E. Cudkowicz, MD Seelos Therapeutics, Inc. · collab

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. Regimen E will evaluate the safety and efficacy of a single study drug, SLS-005 (Trehalose injection, 90.5 mg/mL for intravenous infusion) in participants with ALS.

Timeline

Start: 2022-02-21
Primary completion: 2023-08-25
Completion: 2023-08-25

Outcome

Missed primary endpoint

paper The disease rate ratio for change in ALSFRS-R and survival was 0·87 (95% credible interval 0·665-1·102, posterior probability of superiority 0·877). PMID 40409314 ↗

paper there was no evidence to suggest a difference in ALS disease progression compared with placebo in this study. PMID 40409314 ↗

Publications

HEALEY ALS Platform Trial; HEALEY ALS Platform Trial Study Group. Safety and efficacy of trehalose in amyotrophic lateral sclerosis (HEALEY ALS Platform Trial): an adaptive, phase 2/3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2025 Jun;24(6):500-511. doi: 10.1016/S1474-4422(25)00173-5.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Trehalose	Small molecule	0.75 g/kg	Intravenous