This study is designed to evaluate whether a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis (ALS). Researchers will assess changes in disease progression using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard tool for measuring daily functioning in people with ALS.
The main question is whether taking NAC together with EH-301 can prevent symptom worsening and possibly improve existing ALS symptoms.
Participants will be randomly assigned to receive either the active combination (NAC + EH-301) or matching placebos for 6 months. During this period, they will attend regular clinic visits for evaluations, tests, and safety monitoring.
After completing the initial 6-month phase, all participants may choose to join a 6-month open-label extension, where everyone receives the active treatment regardless of their original group.
Patients diagnosed with ALS according to the Gold Coast criteria;
Disease duration ≤ 18 months;
Men and women aged 18 to 75 years;
Total ALSFRS-R score ≥ 30 for all 12 categories;
Presence of other neurodegenerative diseases;
Significant cognitive impairment and/or dementia;
Any psychiatric illness that could interfere with the study;
Use of dietary supplements with high doses of vitamin B3 in the 30 days prior to inclusion in the study;
Parallel
Acetylcysteine
DrugDaily dose of 600mg of effervescent powder
EH301
Dietary supplementDaily dose of 1800mg of capsules
Riluzole
DrugDaily dose of 100mg of tablets
ALSFRS-R scale
Change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) \[range from 0 to 48, being 48 normal function and 0 complete function loss\] to assess disease progression and evaluate the clinical efficacy and safety of the combination of NAC and EH301 during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Muscle strength
Muscle strength measured by hand dynamometry during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Ventilation-free survival
Percentage of participants who remain alive without requiring tracheotomy or permanent invasive ventilation during the randomized period (24 weeks) or, for those who enter the open-label extension, up to 48 weeks.
Quality of life (ALSAQ-40)
Quality of life assessed using the ALSAQ-40 questionnaire \[range from 0 to 100, being 0 best health status and 100 worst health status\] during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
Respiratory function (%FVC)
Respiratory function measured as percent predicted forced vital capacity (%FVC) during the randomized treatment period (24 weeks) and, for participants who enter the open-label extension, up to 48 weeks.
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