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Trials

Tailoring patients’ enrollment in ALS clinical trials: the effect of disease duration and vital capacity cutoffs

, , , , ORCID Icon, , , , , , , , , , & ORCID Icon show all
Pages 108-115 | Received 15 Mar 2021, Accepted 24 May 2021, Published online: 06 Aug 2021

References

  • Van Eijk RPA, Kliest T, van den Berg LH. Current trends in the clinical trial landscape for amyotrophic lateral sclerosis. Curr Opin Neurol. 2020;33:655–61.
  • Jaiswal MK. Riluzole and edaravone: A tale of two amyotrophic lateral sclerosis drugs. Med Res Rev. 2019;39:733–48.
  • Chiò A, Mazzini L, Mora G. Disease-modifying therapies in amyotrophic lateral sclerosis. Neuropharmacology 2020;167:107986.
  • Westeneng HJ, Debray TPA, Visser AE, van Eijk RPA, Rooney JPK, Calvo A, et al. Prognosis for patients with amyotrophic lateral sclerosis: development and validation of a personalised prediction model. Lancet Neurol. 2018;17:423–33.
  • Steinbach R, Prell T, Gaur N, Stubendorff B, Roediger A, Ilse B, et al. Triage of amyotrophic lateral sclerosis patients during the COVID-19 pandemic: an application of the D50 model. JCM. 2020;9:2873.
  • Calvo A, Vasta R, Moglia C, Matteoni E, Canosa A, Mattei A, et al. Prognostic role of slow vital capacity in amyotrophic lateral sclerosis. J Neurol. 2020;267:1615–21.
  • U.S. Department of Health and Human Services Food and Drug Administration. Amyotrophic lateral sclerosis: developing drugs for treatment. Guidance for industry. September 2019. Available at: https://www.fda.gov/media/130964/download.
  • van den Berg LH, Sorenson E, Gronseth G, Macklin EA, Andrews J, Baloh RH, Airlie House ALS Clinical Trials Guidelines Group, et al. Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials. Neurology. 2019;92:e1610–e1623.
  • Brooks BR, Miller RG, Swash M, Munsat TL, World Federation of Neurology Research Group on Motor Neuron DiseasesEl Escorial revisited: revised criteria for the diagnosis of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord. 2000;1:293–9.
  • Chiò A, Mora G, Leone M, Mazzini L, Cocito D, Giordana MT, Piemonte and Valle d'Aosta Register for ALS (PARALS), et al. Early symptom progression rate is related to ALS outcome: a prospective population-based study. Neurology 2002;59:99–103.
  • McDermott CJ. Clinical trials in amyotrophic lateral sclerosis. Curr Opin Neurol. 2019;32:758–63.
  • Mitsumoto H, Brooks BR, Silani V. Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved? Lancet Neurol. 2014;13:1127–38.
  • Fiala M, Mizwicki MT, Weitzman R, Magpantay L, Nishimoto N. Tocilizumab infusion therapy normalizes inflammation in sporadic ALS patients. Am J Neurodegener Dis 2013;2:129–39.
  • Writing Group; Edaravone (MCI-186) ALS 19 Study Group. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol 2017;16:505–12.
  • Mora JS, Genge A, Chio A, Estol CJ, Chaverri D, Hernández M, on behalf of the AB10015 STUDY GROUP, et al. AB10015 STUDY GROUP. Masitinib as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomized clinical trial. Amyotroph Lateral Scler Frontotemporal Degener. 2020;21:5–14.
  • Paganoni S, Macklin EA, Hendrix S, Berry JD, Elliott MA, Maiser S, et al. Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med. 2020;383:919–30.
  • Berry JD, Taylor AA, Beaulieu D, Meng L, Bian A, Andrews J, Keymer M, et al. Improved stratification of ALS clinical trials using predicted survival. Ann Clin Transl Neurol. 2018;5:474–85.
  • Messina P, Beghi E. Modeling drop-outs in amyotrophic lateral sclerosis. Contemp Clin Trials. 2012;33:218–22.
  • Schulz KF, Grimes DA. Sample size calculations in randomised trials: mandatory and mystical. Lancet. 2005;365:1348–53.

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