Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral...

Area of Science:

• Neuroscience
• Clinical Pharmacology
• Biomarkers

Background:

• Expanded Access Protocols (EAPs) enable patient access to investigational treatments outside of clinical trials.
• EAPs can run in parallel with Randomized Clinical Trials (RCTs), generating valuable real-world evidence.
• This study details a National Institutes of Health (NIH)-funded EAP for amyotrophic lateral sclerosis (ALS).

Purpose of the Study:

• To evaluate the safety and efficacy of trehalose in ALS patients via an EAP.
• To establish a framework for multi-center EAPs that complement RCT data.
• To make collected data and samples available for further research.

Main Methods:

• Seventy participants with ALS were enrolled across 20 US study centers.
• Participants received trehalose for up to 24 weeks.
• Clinical data and biomarkers, including neurofilament light chain and ALSFRS-R scores, were collected.

Main Results:

• Trehalose treatment did not significantly impact neurofilament light chain levels.
• No significant effect on disease progression, measured by ALSFRS-R and slow vital capacity, was observed.
• No unexpected treatment-related risks were identified; serious adverse events were unrelated to trehalose.

Conclusions:

• The EAP successfully established a framework for multi-center expanded access.
• Data generated from this EAP can supplement RCT findings for clinical practice.
• Further NIH-funded EAPs are ongoing, and study data/samples are available for research.