An Exploratory Trial of EPI-589 in Amyotrophic Lateral Sclerosis (EPIC-ALS): Protocol for a Multicenter,...

Area of Science:

• Neuroscience
• Pharmacology
• Clinical Trials

Background:

• Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease with limited treatment options.
• Current ALS drugs like riluzole and edaravone have shown minimal therapeutic benefits.
• EPI-589, a novel redox-active agent, demonstrates potent protective effects against oxidative stress and high blood-brain barrier permeability, suggesting therapeutic potential for ALS.

Purpose of the Study:

• To describe the design and rationale of the EPI-589 Early Phase 2 Investigator-Initiated Clinical Trial for ALS (EPIC-ALS).
• To evaluate the safety and tolerability of EPI-589 in patients diagnosed with ALS.

Main Methods:

• An open-label, single-arm Phase 2 clinical trial (EPIC-ALS) involving patients with ALS.
• A 24-week treatment period with EPI-589 (500 mg three times daily) following a 12-week run-in and preceding a 4-week follow-up.
• Assessment of safety, tolerability, functional changes (ALS Functional Rating Scale-Revised), and various biomarkers (plasma, CSF, MRI) related to oxidative stress and neuronal damage.

Main Results:

• Data collection for the EPIC-ALS trial commenced in September 2021.
• The trial is anticipated to conclude data collection by October 2023.
• Results will provide insights into the safety and potential efficacy of EPI-589 in ALS patients.

Conclusions:

• The EPIC-ALS study is expected to yield valuable data on the characteristics of EPI-589.
• Findings will contribute to understanding EPI-589's potential as a therapeutic agent for ALS.
• This research may inform future clinical development of EPI-589 for neurodegenerative disorders.