Randomized Phase IIIII Study of Acetyl-L-carnitine Effects on Function and Survival in Adults with Amyotrophic Lateral Sclerosis

What this Trial Is About

Researchers are conducting a phase IIIII randomized, double-blind, placebo-controlled trial to study acetyl-L-carnitine ALCAR in adults living with amyotrophic lateral sclerosis ALS. The main goal is to evaluate how two different doses of ALCAR 1.5 gday and 3 gday affect the progression of functional disability, measured by the ALSFRS-R scale. Secondary goals include assessing its impact on lung function, quality of life, cognitive ability, survival without tracheostomy, and various disease-related biomarkers. The study also aims to monitor the safety and tolerability of ALCAR. Participants will be randomly assigned to receive either 1.5 gday or 3 gday of ALCAR, or a placebo, all in addition to riluzole 50 mg twice daily. The treatment will be given three times daily in two pockets for 48 weeks. After enrollment, participants will have follow-up visits at weeks 4, 12, 24, 36, and 48. At each visit, assessments will include vital signs, neurological exams, adverse event monitoring, and blood samples for biomarker analysis. During the study, researchers will assess functional disability regularly using the ALSFRS-R scale and measure respiratory function with spirometry. Cognitive function and quality of life will be evaluated at baseline, week 24, and week 48. Compliance will be checked by counting unused medication packages. Participants will be followed until death, with the main outcome focusing on maintaining self-sufficiency after 48 weeks. Statistical analyses will compare results across treatment groups to evaluate ALCARs effects.

Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS