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 Malentacchi; Mariagrazia Buratti; Paolo 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; Andrea Calvo; Maria Merello; Stefano Messina; 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

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 MalentacchiMariagrazia Buratti, Paolo 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, Andrea Calvo, Maria Merello, Stefano Messina, 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

Risultato della ricerca: Article › peer review

54 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 originale	English
pagine (da-a)	1-9
Numero di pagine	9
Rivista	Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Volume	0(0)
Stato di pubblicazione	Published - 2013

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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.

Valentino, F , La Bella, V, Mancardi, GL, 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, ML, Malentacchi, M, Buratti, M, Messina, P, Tavernelli, F, Papetti, L, Buratti, M, Casa, S, Merello, M, Mennini, T, Giordano, L, Fuda, G, Mascolo, M, Maestri, E, Perini, M, Tremolizzo, L, Giussani, G, Verde, F, Calvo, A, Merello, M, Messina, S, Buzzi, P, Maderna, L, Frasca, V, Bonito, V, Caponnetto, C, Marzorati, L, Lorusso, L, Mazzini, L, Simone, IL, Beghi, E, Logroscino, G, Calvo, A, Ferrarese, C, Bendotti, C, Silani, V, Chiò, A, Messina, S, Corbo, M, Mora, G, Giannini, F, Lunetta, C & Floriani, I 2013, 'Randomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALS.', Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol. 0(0), pagg. 1-9.

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title = "Randomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALS.",

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.",

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

year = "2013",

language = "English",

volume = "0(0)",

pages = "1--9",

journal = "Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration",

issn = "2167-8421",

publisher = "Informa Healthcare",

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TY - JOUR

T1 - Randomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALS.

AU - Valentino, Francesca

AU - La Bella, Vincenzo

AU - Mancardi, Giovanni L.

AU - D'Errico, Eustachio

AU - Marinou, Kalliopi

AU - Samarelli, Vito

AU - Moglia, Cristina

AU - Valentino, Francesca

AU - Ticozzi, Nicola

AU - Cavallo, Enrico

AU - Cammarosano, Stefania

AU - Tortelli, Rosanna

AU - Giacomelli, Elena

AU - Testa, Lucia

AU - Rizzo, Aurora

AU - Pupillo, Elisabetta

AU - Inghilleri, Maurizio

AU - Morelli, Cludia

AU - Quadrelli, Maria Lidia

AU - Malentacchi, Maria

AU - Buratti, Mariagrazia

AU - Messina, Paolo

AU - Tavernelli, Fiorella

AU - Papetti, Laura

AU - Buratti, Mariagrazia

AU - Casa, Stefania

AU - Merello, Maria

AU - Mennini, Tiziana

AU - Giordano, Laura

AU - Fuda, Giuseppe

AU - Mascolo, Maria

AU - Maestri, Eleonora

AU - Perini, Michele

AU - Tremolizzo, Lucio

AU - Giussani, Giorgia

AU - Verde, Federico

AU - Calvo, Andrea

AU - Merello, Maria

AU - Messina, Stefano

AU - Buzzi, Paolo

AU - Maderna, Luca

AU - Frasca, Vittorio

AU - Bonito, Virginio

AU - Caponnetto, Claudia

AU - Marzorati, Laura

AU - Lorusso, Lorenzo

AU - Mazzini, Letizia

AU - Simone, Isabella L.

AU - Beghi, Ettore

AU - Logroscino, Giancarlo

AU - Calvo, Andrea

AU - Ferrarese, Carlo

AU - Bendotti, Caterina

AU - Silani, Vincenzo

AU - Chiò, Adriano

AU - Messina, Stefano

AU - Corbo, Massimo

AU - Mora, Gabriele

AU - Giannini, Fabio

AU - Lunetta, Christian

AU - Floriani, Irene

PY - 2013

Y1 - 2013

N2 - 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.

AB - 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.

UR - http://hdl.handle.net/10447/71824

M3 - Article

SN - 2167-8421

VL - 0(0)

SP - 1

EP - 9

JO - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

JF - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

ER -

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

Abstract

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