1. Randomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALS
- Author
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Beghi, E, Pupillo, E, Bonito, V, Buzzi, P, Caponnetto, C, Chiò, A, Corbo, M, Giannini, F, Inghilleri, M, Bella, Vl, Logroscino, G, Lorusso, L, Lunetta, C, Mazzini, L, Messina, P, Mora, G, Perini, M, Quadrelli, Ml, Silani, V, Simone, Il, Tremolizzo, L, Samarelli, V, Tortelli, R, D'Errico, E, Merello, M, Tavernelli, F, Mancardi, GIOVANNI LUIGI, Mascolo, M, Bendotti, C, Buratti, M, Floriani, I, Giordano, L, Giussani, G, Maderna, L, Maestri, E, Marinou, K, Mennini, T, Messina, S, Morelli, C, Papetti, L, Rizzo, A, Ticozzi, N, Verde, F, Ferrarese, C, Marzorati, L, Testa, L, Valentino, F, Frasca, V, Giacomelli, E, Casali, S, Malentacchi, M, Calvo, A, Cammarosano, S, Moglia, C, Cavallo, E, Fuda, G., Beghi,E, Pupillo, E, Bonito, V, Buzzi, P, Caponnetto, C, Chiò, A, Corbo, M, Giannini, F, Inghilleri, M, La Bella, V, Logroscino,G, Lorusso,L, Lunetta,C, Mazzini, L, Messina, P, Mora, G, Perini, M, Quadrelli, ML, Silani, V, Simone, IL, Tremolizzo, L, Valentino, F, The Italian ALS, Study Group, Beghi, E, Bella, V, Logroscino, G, Lorusso, L, Lunetta, C, Quadrelli, M, Simone, I, The Italian ALS Study Group, I, and Ferrarese, C
- Subjects
Male ,amyotrophic lateral sclerosis ,Vital Capacity ,Placebo-controlled study ,Pilot Projects ,Gastroenterology ,law.invention ,Randomized controlled trial ,law ,Acetyl-L-carnitine ,motor neuron disease ,randomized trial ,acetyl-l-carnitine ,Amyotrophic lateral sclerosis ,Acetylcarnitine ,ALS, acetyl-L-carnitine ,Nootropic Agents ,Riluzole ,Middle Aged ,Treatment Outcome ,Neurology ,Combination ,Disease Progression ,Drug Therapy, Combination ,Settore MED/26 - Neurologia ,Female ,medicine.drug ,Adult ,medicine.medical_specialty ,Acetyl-L-carnitine, amyotrophic lateral sclerosis, motor neuron disease, randomized trial ,Double blind ,Double-Blind Method ,Drug Therapy ,Internal medicine ,medicine ,Humans ,Aged ,MED/26 - NEUROLOGIA ,business.industry ,Disease progression ,medicine.disease ,Surgery ,Quality of Life ,Amyotrophic Lateral Sclerosis ,Excitatory Amino Acid Antagonists ,Neurology (clinical) ,business - 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.
- Published
- 2013