Randomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALS

Ettore Beghi, Elisabetta Pupillo, Virginio Bonito, Paolo Buzzi, Claudia Caponnetto, Adriano Chiò, Massimo Corbo, Fabio Giannini, Maurizio Inghilleri, Vincenzo La Bella, Giancarlo Logroscino, Lorenzo Lorusso, Christian Lunetta, Letizia Mazzini, Paolo Messina, Gabriele Mora, Michele Perini, Maria Lidia Quadrelli, Vincenzo Silani, Isabella L. SimoneLucio Tremolizzo, Vito Samarelli, Rosanna Tortelli, Eustachio D'Errico, Maria Merello, Fiorella Tavernelli, Giovanni L. Mancardi, Maria Mascolo, Caterina Bendotti, Mariagrazia Buratti, Irene Floriani, Laura Giordano, Giorgia Giussani, Luca Maderna, Eleonora Maestri, Kalliopi Marinou, Tiziana Mennini, Stefano Messina, Cludia Morelli, Laura Papetti, Aurora Rizzo, Nicola Ticozzi, Federico Verde, Carlo Ferrarese, Laura Marzorati, Lucia Testa, Francesca Valentino, Vittorio Frasca, Elena Giacomelli, Stefania Casa, Maria Malentacchi, Andrea Calvo, Stefania Cammarosano, Cristina Moglia, Enrico Cavallo, Giuseppe Fuda

Research output: Contribution to journalArticlepeer-review


Our objective was to assess the effects of acetyl-L-carnitine (ALC) with riluzole on disability and mortality of amyotrophic lateral sclerosis (ALS). Definite/probable ALS patients, 40-70 years of age, duration 6-24 months, self-sufficient (i.e. able to swallow, cut food/handle utensils, and walk), and with forced vital capacity (FVC) > 80% entered a pilot double-blind, placebo-controlled, parallel group trial and were followed for 48 weeks. ALC or placebo 3 g/day was added to riluzole 100 mg/day. Primary endpoint: number of patients no longer self-sufficient. Secondary endpoints: changes in ALSFRS-R, MRC, FVC and McGill Quality of Life (QoL) scores. Analysis was made in the intention-to-treat (ITT) and per-protocol (PP) population, completers and completers/compliers (i.e. taking > 75% of study drug). Forty-two patients received ALC and 40 placebo. In the ITT population, 34 (80.9%) patients receiving ALC and 39 (97.5%) receiving placebo became non-self-sufficient (p = 0.0296). In the PP analysis, percentages were 84.4 and 100.0% (p = 0.0538), respectively. Mean ALSFRS-R scores at 48 weeks were 33.6 (SD 10.4) and 27.6 (9.9) (p = 0.0388), respectively, and mean FVC scores 90.3 (32.6) and 58.6 (31.2) (p = 0.0158), respectively. Median survival was 45 months (ALC) and 22 months (placebo) (p = 0.0176). MRC, QoL and adverse events were similar. In conclusion, ALC may be effective, well-tolerated and safe in ALS. A pivotal phase III trial is needed.

Original languageEnglish
Pages (from-to)397-405
Number of pages9
JournalAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Issue number5-6
Publication statusPublished - Sep 2013


  • Acetyl-L-carnitine
  • Amyotrophic lateral sclerosis
  • Motor neuron disease
  • Randomized trial

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology


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