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Review Article

Clinical trials in ALS: A review of the role of clinical and neurophysiological measurements

, &
Pages 202-212 | Received 07 Mar 2005, Accepted 05 Jul 2005, Published online: 10 Jul 2009

References

  • Turner M. R., Parton M. J., Leigh P. N. Clinical trials in ALS: an overview. Sem Neurol 2001; 21: 167–75
  • Miller R. G., Munsat T. L., Swash M., Brooks B. R. Consensus guidelines for the design and implementation of clinical trials in ALS. World Federation of Neurology committee on research. J Neurol Sci 1999; 169: 2–12
  • Matthews J. N. S., ed. An Introduction to Randomized Clinical Trials. Arnold, London 2000
  • Swash M. Early diagnosis of ALS/MND. J Neurol Sci 1998; 160((Suppl))S33–6
  • Appel S. H., Stewart S. S., Appel V., Harati Y., Mietlowski W., Weiss W., Belendiuk G. W. A double‐blind study of the effectiveness of cyclosporine in amyotrophic lateral sclerosis. Arch Neurol 1988; 45: 381–6
  • The Italian ALS Study Group. Branched‐chain amino acids and amyotrophic lateral sclerosis: a treatment failure?. Neurology 1993; 43: 2466–70
  • Parton M., Mitsumoto H., Leigh P. N. Amino acids for amyotrophic lateral sclerosis/motor neuron disease. The Cochrane Library, Issue 4. John Wiley & Sons, Ltd, ChichesterUK 2004
  • Smith R. A., Melmed S., Sherman B., Frane J., Munsat T. L., Festoff B. W. Recombinant growth hormone treatment of amyotrophic lateral sclerosis. Muscle Nerve 1993; 16: 624–33
  • Drachman D. B., Chaudhry V., Cornblath D., Kuncl R. W., Pestronk A., Clawson L., et al. Ann Neurol 1994; 35: 142–50
  • Bensimon G., Lacomblez L., Meininger V., and the ALS/Riluzole study group. A controlled trial of riluzole in amyotrophic lateral sclerosis. New England Journal of Medicine 1994; 330: 585–91
  • Miller R. G., Mitchell J. D., Moore D. H. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). (Cochrane Review). The Cochrane Library, Issue 4, 2004. John Wiley & Sons, Ltd, ChichesterUK
  • Louwerse E. S., Weverling G. J., Bossuyt P. M., Meyjes F. E., de Jong J. M. Randomized, double‐blind, controlled trial of acetylcysteine in amyotrophic lateral sclerosis. Arch Neurol 1995; 52: 559–64
  • Orrell R. W., Lane J. M., Ross M. A. Antioxidant treatment for amyotrophic lateral sclerosis/motor neuron disease (Cochrane Review). The Cochrane Library, Issue 4. John Wiley & Sons, Ltd, ChichesterUK 2004
  • ALS CNTF Treatment Study Group. A double‐blind placebo‐controlled clinical trial of subcutaneous recombinant human ciliary neurotrophic factor (rHCNTF) in amyotrophic lateral sclerosis. Neurology 1996; 46: 1244–9
  • Bongioanni P., Reali C., Sogos V. Ciliary neurotrophic factor (CNTF) for amyotrophic lateral sclerosis/motor neuron disease (Cochrane Review). The Cochrane Library, Issue 4. John Wiley & Sons, Ltd, ChichesterUK 2004
  • Miller R. G., Petajan J. H., Bryan W. W., Armon C., Barohn R. J., Goodpasture J. C., , and the rhCNTF ALS ALS Study Group, et al. A placebo‐controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis. Ann Neurol 1996; 39: 256–60
  • Tandan R., Bromberg M. B., Forshew R. N., Fries T. J., Badger G. J., Carpenter J., et al. A controlled trial of amino acid therapy in amyotrophic lateral sclerosis. I. Clinical, functional, and maximum isometric torque data. Neurology 1996; 47: 1220–6
  • Miller R. G., Moore D., Young L. A., Armon C., Barohn R. J., Bromberg M. B., Bryan W. W., et al. Placebo‐controlled trial of gabapentin in patients with amyotrophic lateral sclerosis. WALS Study Group. Western Amyotrophic Lateral Sclerosis Study Group. Neurology 1996; 47: 1383–8
  • Lacomblez L., Bensimon G., Leigh P. N., Guillet P., Meininger V. Dose‐ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis/Riluzole Study Group II. Lancet 1996; 25(347)1425–31
  • Lai E. c., Felice K. J., Festoff B. W., Gawel M. J., Gelinas D. F., Kratz R., et al. and the North America ALS/IGF‐I Study Group. Effects of recombinant human insulin‐like growth factor‐I on progression of ALS. A placebo controlled study. Neurology 1997; 49: 1621–30
  • Mitchell J. D., Wokke J. H. J., Borasio G. D. Recombinant human insulin‐like growth factor I (rhIGF‐I) for amyotrophic lateral sclerosis/motor neuron disease (Cochrane Review). The Cochrane Library, Issue 4. John Wiley & Sons, Ltd, ChichesterUK 2004
  • Lange D. J., Murphy P. L., Diamond B., Appel V., Lai E. C., Younger D. S., Appel S. H. Selegiline is ineffective in a collaborative double‐blind, placebo‐controlled trial for treatment of amyotrophic lateral sclerosis. Arch Neurol 1998; 55: 93–6
  • Borasio G. D., Robberecht W., Leigh P. N., Emile J., Guilloff R. J., Jerusalem F., et al. and the European ALS/IGF‐I Study Group. A placebo‐controlled trial of insulin‐like nerve growth factor‐1 in amyotrophic lateral sclerosis. Neurology 1998; 51: 583–6
  • The BDNF Study Group (Phase III). A controlled trial of recombinant methionyl human BDNF in ALS. Neurology 1999; 52: 1427–33
  • Beghi E., Chiò A., Inghilleri M., Mazzini L., Micheli A., Mora G., et al. and the Italian Amyotrophic Lateral Sclerosis Study Group. A randomized controlled trial of recombinant interferon beta‐1a in ALS. Neurology 2000; 54: 469–74
  • Miller R. G., Moore D. H., Gelinas D. F., Dronsky V., Mendoza M., Barohn R. J., et al. and the Western ALS Study Group. Phase III randomized trial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology 2001; 56: 843–8
  • Desnuelle C., Dib M., Garrel C., Favier A. A double‐blind, placebo‐controlled randomized clinical trial of alpha‐tocopherol (vitamin E) in the treatment of amyotrophic lateral sclerosis. ALS riluzole‐tocopherol Study Group. Amyotroph Lateral Scler Other Motor Neuron Disord 2001; 2: 9–18
  • Groeneveld G. J., Veldink J. H., van der Tweel I., Kalmijn S., Beijer C., de Visser M., et al. A randomized sequential trial of creatine in amyotrophic lateral sclerosis. Ann Neurol 2003; 53: 437–45
  • Cudkowicz M. E., Shefner J. M., Schoenfeld D. A., Brown RH J. r., Johnson H., Qureshi M., et al. and the Northeast ALS Consortium. A randomized, placebo‐controlled trial of topiramate in amyotrophic lateral sclerosis. Neurology 2003; 61: 456–64
  • Meiningier V., Bensimon G., Bradley W. G., Brooks B. R., Doillet P., Eisen A. A., et al. Efficacy and safety of xaliproden in amyotrophic lateral sclerosis: results of two phase III trials. Amyotroph Lateral Scler Other Motor Neuron Disord 2004; 5: 107–17
  • Graf M., Ecker D., Horowski R., Kramer B., Riederer P., Gerlach M., et al. on behalf of the German vitamin E/ALS study group. High dose vitamin E therapy in amyotrophic lateral sclerosis as add‐on therapy to riluzole: results of a placebo‐controlled double‐blind study. J Neural Transm 2004, published on‐line October 27
  • Shefner J. M., Cudkowicz E., shoenfeld D., Conrad T., Taft J., Chilton M., et al. and the NEALS Consortium. A clinical trial of creatine in ALS. Neurology 2004; 63: 1656–61
  • de Carvalho M., Scotto M., Lopes A., Swash M. Clinical and neurophysiological evaluation of progression in amyotrophic lateral sclerosis. Muscle Nerve 2003; 28: 630–3
  • de Carvalho M., Scotto M., Lopes A., Swash M. Quantifying progression in ALS. Neurology 2005; 64: 1783–5
  • Survival Cedarbaum J. Amyotroph Lateral Scler Other Motor Neuron Disord. 2004; 5((Suppl 1))79–83
  • Pinto A. C., Evangelista T., de Carvalho M., Alves M., Sales‐Luís M. L. Respiratory assistance with non‐invasive ventilation (Bipap) in MND/ALS patients: survival rate in a controlled trial. J Neurol Sci 1995; 129((Suppl))19–26
  • Desport J. C., Preux P. M., Truong C. T., Courat L., Vallat J. M., Couratier P. Nutritional assessment and survival in ALS patients. Amyotroph Lateral Scler Other Motor Neuron Disord 2000; 1: 91–6
  • Munsat T. L., Andres P. L., Finison L., Conlon T., Thibodeau L. The natural history of motor neuron loss in amyotrophic lateral sclerosis. Neurology 1988; 38: 409–11
  • Lyall R. A., Donaldson N., Polkey M. I., Leigh P. N., Moxham J. Respiratory muscle strength and ventilatory failure in amyotrophic lateral sclerosis. Brain 2001; 124: 2000–13
  • Fitting J. W., Paillex R., Hirt L., Aebischer P., Schluep M. Sniff nasal pressure: a sensitive respiratory test to assess progression of amyotrophic lateral sclerosis. Ann Neurol 1999; 46: 887–93
  • Norris FH J. r., Calanchini P. R., Fallat R. J., Panchari S., Jewett B. The administration of guanidine in amyotrophic lateral sclerosis. Neurology 1974; 24: 721–8
  • Appel V., Stewart S. S., Smith G., Appel S. H. A rating scale for amyotrophic lateral sclerosis: description and preliminary experience. Ann Neurol 1987; 22: 328–33
  • Cedarbaum J. M., Stambler N. Performance of Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) in multicenter clinical trials. J Neurol 1997; 152((Suppl 1))S1–9
  • Traynor B. J., Zhang H., Shefner J. M., Schoenfeld D., Cudkowicz M., and EALS Consortium. Functional outcome measures as clinical trial endpoints in ALS. Neurology 2004; 63: 1933–5
  • Swash M. Health outcome and quality‐of‐life measurements in amyotrophic lateral sclerosis. J Neurol 1997; 244((Suppl 2))S26–9
  • Miller R. G., Moore D. H. ALS trial design: expectation and reality. Amyotroph Lateral Scler Other Motor Neuron Disord 2004; 5((Suppl 1))52–4
  • Cedarbaum J. M., Stambler N., Malta E., Fuller C., Hilt D., Thurmond B., Nakanishi A. The ALS‐FRS: a revised ALS functional rating scale that incorporates assessments of respiratpry function. BNDF ALS Study Group (phase II/III). J Neurol Sci 1999; 169: 13–21
  • Swash M., de Carvalho M. The neurophysiological índex in ALS. Amyotroph Lateral Scler Other Motor Neuron Disord 2004; 5((Suppl 1))10810
  • de Carvalho M., Chio A., Dengler R., Hecht M., Weber M., Swash M. Neurophysiological measures in amyotrophic lateral sclerosis: markers of progression in clinical trials. Amyotroph Lateral Scler Other Motor Neuron Disord., in press
  • Swash M. What does the neurologist expect from clinical neurophysiology?. Muscle Nerve 2002; 11(Suppl)S1348
  • Fareed G. C., Tyler R. The use of isoprinosine in patients with amyotrophic lateral sclerosis. Neurology 1971; 21: 937–40
  • Olson W. H., Simons J. A., Halaas G. W. Therapeutic trial of tilorone: lack of benefit in a double‐blind, placebo‐controlled study. Neurology 1978; 28: 1293–5
  • Munsat T. L., Easterday C. S., Levy S., Wolff S. M., Hiatt R. Amantadine and guanidine are ineffective in ALS. Neurology 1981; 31: 1054–5
  • Bradley W. G. Double‐blind controlled trial of purified brain gangliosides in amyotrophic lateral sclerosis and experience with peripheral neuropathies. Adv Exp Med Biol (1984); 174: 565–73
  • Bradley W. G., Hedlund W., Cooper C., Desousa G. J., Gabbai A., Mora J. S., et al. A double‐blind controlled trial of bovine brain gangliosides in amyotrophic lateral sclerosis. Neurology 1984; 34: 1079–82
  • Harrington H., Hallett M., Tyler H. R. Gangliosides therapy for amyotrophic lateral sclerosis: a double‐blind controlled trial. Neurology 1984; 34: 1083–5
  • Hallett M., Harrington H., Tyler H. R., Flood T., Slater N. Trials of gangliosides for amyotrophic lateral sclerosis and diabetic neuropathy. Adv Exp Med Biol 1984; 174: 575–9
  • Imoto K., Saida K., Iwamura K., Saida T., Nishitani H. Amyotrophic lateral sclerosis: a double‐blind crossover trial of thyrotropin‐releasing hormone. J Neurol Neurosurg Psychiatry 1984; 47: 1332–4
  • Olarte M. R., Shafer S. Q. Levamisole is ineffective in the treatment of amyotrophic lateral sclerosis. Neurology 1985; 35: 1063–6
  • Mitsumoto H., Salgado E. D., Negroski D., Hanson M. R., Salanga V. D., Wilber J. F., et al. Amyotrophic lateral sclerosis: effects of acute intravenous and chronic subcutaneous administration of thyrotropin‐releasing hormone in controlled trials. Neurology 1986; 36: 152–9
  • Brooke M. H., Florence J. M., Heller S. L., Kaiser K. K., Phillips D., Gruber A., et al. Controlled trial of thyrotropin releasing hormone in amyotrophic lateral sclerosis. Neurology 1986; 36: 146–51
  • Caroscio J. T., Cohen J. A., Zawodniak J., Takai V., Shapiro A., Blaustein S., et al. A double‐blind, placebo‐controlled trial of TRH in amyotrophic lateral sclerosis. Neurology 1986; 36: 141–5
  • Norris F. H., Denys E. H., Fallat R. J. Trial of octaconasol in amyotrophic lateral sclerosis. Neurology 1986; 36: 1263–4
  • Plaitakis A., Smith J., Mandeli J., Yahr M. D. Pilot trial of branched‐chain amino acids in amyotrophic lateral sclerosis. Lancet 1988; 1: 1015–8
  • Lacomblez L., Bouche P., Bensimon G., Meininger V. A double‐blind, placebo‐controlled trial of high doses of gangliosides in amyotrophic lateral sclerosis. Neurology 1989; 39: 1635–7
  • Blin O., Serratrice G., Pouget J., Aubrespy G., Guelton C., Crevat A. Short‐term double‐blind vs. placebo trial of L‐threonine in amyotrophic lateral sclerosis. Presse Med 1989; 30: 1469–70
  • Testa D., Caraceni T., Fetoni V. Branched‐chain amino acids in the treatment of amyotrophic lateral sclerosis. J. Neurol 1989; 236: 445–7
  • Blin O., Desnuelle C., Guelton C., Aubrespy G., Ardissonne J. P., Crevat A., et al. Anomalie des acides amines neurotransmetteurs dans la slerose lateral amyotrophique: une application therapeutique. Rev Neurol 1991; 147: 392–4
  • Blin O., Pouget J., Aubrespy G., Guelton C., Crevat A., Serratrice G. A double‐blind placebo‐controlled trial of L‐threonine in amyotrophic lateral sclerosis. J. Neurol 1992; 239: 79–81
  • Gil R., Neau J. P., Courtois P., Gaucher C., Jonveaux T., Rosolacci T., et al. Essai en double aveugle contre placebo des acides amines branches et de la L‐threonine dans le traitment des signes et symptomes de la sclerose lateral amyotrophique sur un courte periode. Sem Hop Paris 1992; 68: 1472–5
  • Testa D., Caraceni T., Fetoni V., Girotti. Chronic treatment with L‐threonine in amyotrophic lateral sclerosis: a pilot study. Clin Neurol Neurosurg 1992; 94: 7–9
  • Blin O., Pouget J., Aubrespy G., Guelton C., Crevat A., Serratrice G. A double‐blind placebo‐controlled trial of L‐threonine in amyotrophic lateral sclerosis. J Neurol 1992; 239: 79–81
  • Askmark H., Aquilonius S. M., Gillberg P. G., Liedholm L. J., Stalberg E., Wuopio R. A pilot trial of dextromethorphan in amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry 1993; 56: 197–200
  • Hesselmans L. F., Wieneke G. H., Oey P. L., Groenhout D. M., van der Graaf Y., Gispen W. H., Jennekens F. G. Evaluation of electrophysiological and clinical tests in an exploratory trial of Org 2766 in motor neuron disease. Neuromuscul Disord 1993; 3: 319–25
  • Eisen A., Stewart H., Schulzer M., Cameron D. Anti‐glutamate therapy in amyotrophic lateral sclerosis. Can J Neurol Sci 1993; 20: 297–301
  • Mazzini L., Testa D., Balzarini C., Mora G. An open.randomized clinical trial of selegiline in amyotrophic lateral sclerosis. J Neurol 1994; 241: 223–7
  • Jossan S. S., Ekblom J., Gudjonsson O., Hagbarth K. E., Aquilonius S. M. Double‐blind crossover trial with deprenyl in amyotrophic lateral sclerosis. J Neural Transm 1994; 41: 237–41
  • Miller R. G., Shepherd R., Dao H., Khramstov A., Mendonza M., Graves J., Smith S. Controlled trial of nimodipine in amyotrophic lateral sclerosis. Neuromusc Disord 1996; 6: 101–4
  • Blin O., Azulay J. P., Desnuelle C., Bille‐Turc F., Braguer D., Besse D., et al. A controlled one‐year trial of dextromethorphan in amyotrophic lateral sclerosis. Clin Neuropharmacol 1996; 19: 189–92
  • Gredal O., Werdelin L., Bak S., Christensen P. B., Boysen G., Kristensen M. O., et al. A clinical trial of dextromethorphan in amyotrophic lateral sclerosis. Acta Neurol Scand 1997; 96: 8–13
  • Kaji R., Kodama M., Imamura A., Hashida T., Kohara N., Ishizu M., et al. Effect of ultra‐high dose methylcobalamin on compound muscle action potentials in amyotrophic lateral sclerosis: a double‐blind controlled study. Muscle Nerve 1998; 21: 1775–8
  • Chio A., Cucatto A., Terreni A. A., Schiffer D. Reduced glutathione in amyotrophic lateral sclerosis: an open, crossover, randomized trial. Ital J Neurol Sci 1998; 19: 363–6
  • Bensimon G., Lacomblez L., Delumeau J. C., Bejuit R., Truffinet P., Meininger V. Riluzole/ALS Study Group. A study of riluzole in the treatment of advanced stage or elderly patients with amyotrophic lateral sclerosis. Journal of Neurology 2002; 249: 609–15
  • Ryberg H., Askmark H., Persson L. I. A double‐blind randomized clinical trial in amyotrophic lateral sclerosis using lamotrigine: effects on CSF glutamate, aspartate, branched‐chain amino acid levels and clinical parameters. Acta Neurol Scand 2003; 108: 1–8
  • Yuen E. C., Olney R. K. Longitudinal study of fibre density and motor unit number estimate in patients with amyotrophic lateral sclerosis. Neurology 1997; 49: 573–8
  • Felice K. J. A longitudinal study comparing thenar motor unit number estimates to other quantitative tests in patients with amyotrophic lateral sclerosis. Muscle Nerve 1997; 20: 179–85
  • Bromberg M. B., Fries T. J., Forshew D. A., Tandan R. Electrophysiological endpoint measures in a multicentre ALS drug trial. J Neurol Sci 2001; 184: 51–5
  • Shefner J. M., Cudkowicz M. E., Zhang H., Schoenfeld D., Jillapalli D., Northeast ALS Consortium. The use of statistical MUNE in a multicentre clinical trial. Muscle Nerve 2004; 30: 463–9

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