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

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

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29 Citazioni (Scopus)

Abstract

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.
Lingua originaleEnglish
pagine (da-a)1-9
Numero di pagine9
RivistaAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Volume0(0)
Stato di pubblicazionePublished - 2013

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All Science Journal Classification (ASJC) codes

  • Neurology
  • Clinical Neurology

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Valentino, F., La Bella, V., Mancardi, G. L., D'Errico, E., Marinou, K., Samarelli, V., Moglia, C., Valentino, F., Ticozzi, N., Cavallo, E., Cammarosano, S., Tortelli, R., Giacomelli, E., Testa, L., Rizzo, A., Pupillo, E., Inghilleri, M., Morelli, C., Quadrelli, M. L., ... Floriani, I. (2013). Randomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALS. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 0(0), 1-9.