Double-Blind Placebo-Controlled Trial on the Use of Acetyl-L-Carnitine for the Treatment of Amyotrophic Lateral Sclerosis (P04.151)
Bibliographic record
Abstract
Objective: To assess superiority of acetyl-L-carnitine/riluzole (ALC) combination over riluzole alone on functional disability in patients with amyotrophic lateral sclerosis (ALS). Background ALS is a fatal motorneuron disease. Unlike riluzole, no disease-modifier drugs are available. ALC counteracts motor neuron death induced by toxic agents or deprivation of trophic factors and reduces disease progression in animal models. Design/Methods: Patients 40-70 years with definite or probable ALS, disease duration 6-24 months, self-supporting (ie, able to swallow, cut foods/handling utensils, and walk), and with forced vital capacity (FVC)>80% were enrolled in a pilot double-blind, placebo-controlled, parallel group trial and followed for 48 weeks. ALC 3g/day and placebo were added to riluzole 100mg/day. Primary end-point: no. patients becoming non self-supporting. Secondary end-points: changes in the ALS-FRS-R scale, MRC scale, FVC and McGill quality-of-life scale. Analysis was performed in the intention-to-treat (ITT) population, completers and completers/ compliers (ie, taking >75% of study drug). Results: 42 patients received ALC and 40 patients placebo. In the ITT population, 34 patients receiving ALC (80.9%) and 39 receiving placebo (97.5%) became non self-supporting (p=0.030). Percentages in study completers were 71.4 vs. 94.7% (p=0.064) and in "completers/compliers", 71.4% vs. 94.4% (p=0.069). In the per-protocol analysis, percentages were 84.4 vs. 100.0% (p=0.054) and 76.2 vs. 100.0% (p=0.132)(completers & completers/compliers). At 48 weeks, ALC was associated with higher mean ALR-FRS-R scores (p=0.039) and FVC scores (p=0.016), while MRC and quality of life were no different. Adverse events were similar. Conclusions: ALC appears superior to placebo and is well-tolerated and safe. A confirmatory phase III trial is needed. (*) Italian ALS Study Group: Virginio Bonito, Paolo Buzzi, Claudia Caponnetto, Adriano Chiò, Massimo Corbo, Fabio Giannini, Maurizio Inghilleri, Vincenzo La Bella, Lorenzo Lorusso, Christian Lunetta, Letizia Mazzini, Paolo Messina, Gabriele Mora, Michele Perini, Elisabetta Pupillo, Maria Lidia Quadrelli, Vincenzo Silani, Isabella Simone, Lucio Tremolizzo.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.002 | 0.002 |
| Meta-epidemiology (narrow) | 0.002 | 0.001 |
| Meta-epidemiology (broad) | 0.005 | 0.001 |
| Bibliometrics | 0.001 | 0.000 |
| Science and technology studies | 0.001 | 0.002 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.003 | 0.003 |
| Insufficient payload (model declined to judge) | 0.009 | 0.002 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".