6533b7d8fe1ef96bd126af1f
RESEARCH PRODUCT
Randomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALS.
E BeghiE PupilloV BonitoP BuzziC CaponnettoA ChiòM CorboF GianniniM InghilleriVl BellaG LogroscinoL LorussoC LunettaL MazziniP MessinaG MoraM PeriniMl QuadrelliV SilaniIl SimoneL TremolizzoV SamarelliR TortelliE D'erricoM MerelloF TavernelliGiovanni Luigi MancardiM MascoloC BendottiM BurattiI FlorianiL GiordanoG GiussaniL MadernaE MaestriK MarinouT MenniniS MessinaC MorelliL PapettiA RizzoN TicozziF VerdeC FerrareseL MarzoratiL TestaF ValentinoV FrascaE GiacomelliS CasaliM MalentacchiA CalvoS CammarosanoC MogliaE CavalloG. Fudasubject
Maleamyotrophic lateral sclerosisVital CapacityPlacebo-controlled studyPilot ProjectsGastroenterologylaw.inventionRandomized controlled triallawAcetyl-L-carnitineamyotrophic lateral sclerosis; motor neuron disease; randomized trial; acetyl-l-carnitinerandomized trialAmyotrophic lateral sclerosisAcetylcarnitineALS acetyl-L-carnitineNootropic AgentsRiluzoleMiddle AgedRiluzoleTreatment OutcomeNeurologyCombinationDisease Progressionmotor neuron diseaseDrug Therapy CombinationSettore MED/26 - NeurologiaFemaleAcetylcarnitinemedicine.drugAdultmedicine.medical_specialtyAcetyl-L-carnitine amyotrophic lateral sclerosis motor neuron disease randomized trialDouble blindDouble-Blind MethodDrug TherapyInternal medicinemedicineHumansAgedMED/26 - NEUROLOGIAbusiness.industryDisease progressionmedicine.diseaseAcetyl-L-carnitineSurgeryQuality of LifeAcetylcarnitine; Adult; Aged; Amyotrophic Lateral Sclerosis; Disease Progression; Double-Blind Method; Drug Therapy Combination; Excitatory Amino Acid Antagonists; Female; Humans; Male; Middle Aged; Nootropic Agents; Pilot Projects; Quality of Life; Riluzole; Treatment Outcome; Vital CapacityNeurology (clinical)businessExcitatory Amino Acid Antagonistsdescription
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.
| year | journal | country | edition | language |
|---|---|---|---|---|
| 2013-01-01 |