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Kaji et al recently conducted a phase II/III clinical trial to evaluate the safety and efficacy of methylcobalamin in 343 patients with amyotrophic lateral sclerosis (ALS).1 Both coprimary endpoints (survival and ALS Functional Rating Scale [ALSFRS-R]) failed to show significant benefit (p=0.19 and p=0.13, respectively). The choice for two coprimary endpoints is interesting and may have circumvented important pitfalls encountered by previous ALS trials.2 As ALS significantly reduces the patient’s life expectancy, evaluating a drug’s therapeutic potential to improve survival is fundamental. Survival time, however, may be influenced by life-extending interventions (eg, gastrostomy or tracheostomy) and provides little information about the patient’s functioning during life. The ALSFRS-R is a welcome alternative that is clinically relevant, easily obtained and highly predictive of survival time. Unfortunately, positive phase II results on the ALSFRS-R have until now …
Contributors RPAvE: study concept, design, analysis, interpretation of data and drafting the manuscript. JR: study concept, design, analysis, interpretation of data and drafting the manuscript. OH: study concept and critical revision of the manuscript for intellectual content. LHvdB: study concept and critical revision of the manuscript for intellectual content.
Funding This study was funded by the Netherlands ALS Foundation (grant number: TRICALS).
Competing interests None declared.
Patient consent for publication Not required.
Provenance and peer review Not commissioned; internally peer reviewed.
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