Pergine Valsugana

Researchers are evaluating acetyl-L-carnitine (ALCAR) in a Phase II/III multicenter, randomized, double-blind, placebo-controlled trial involving 246 adults with amyotrophic lateral sclerosis (ALS). The main goal is to assess how ALCAR, at doses of 1.5 g/day and 3 g/day, affects the progression of functional disability, specifically the loss of self-sufficiency, measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Secondary aims include examining ALCAR's impact on functional decline, respiratory function, quality of life, cognitive function, survival without tracheostomy, and various disease biomarkers related to the drug's action, as well as evaluating safety and tolerability. Participants will be randomly assigned to receive either ALCAR (1.5 or 3 g/day) or placebo alongside riluzole 50 mg twice daily for a treatment period of 48 weeks. The study uses a 1:1:1 randomization scheme with permuted blocks of size 6. Follow-up visits occur at weeks 4, 12, 24, 36, and 48 during which clinical assessments, biomarker testing, and functional evaluations will be conducted. Blood samples for biomarker analysis are collected at baseline and each follow-up visit. Cognitive function and quality of life will be assessed at baseline, week 24, and week 48. Participants will undergo general assessments including vital signs, neurological exams, and monitoring of adverse events at each visit. Functional disability and respiratory function will be evaluated with validated scales and spirometry at specified intervals. Compliance will be monitored by counting unused medication packages. The study will analyze outcomes such as the proportion of self-sufficient participants at 48 weeks, changes in clinical scores, biomarker levels, survival, and safety events, with follow-up continuing until death.