Advanced search
Publication Cover
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Volume 17, 2016 - Issue 5-6
Submit an article Journal homepage
1,263
Views
31
CrossRef citations to date
0
Altmetric
Research Article

A randomized, placebo-controlled, double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis

Jeremy M. ShefnerBarrow Neurological Institute, Phoenix, Arizona and Correspondence[email protected]
,
Andrew A. WolffCytokinetics, Inc., South San Francisco, California, USA
,
Lisa MengCytokinetics, Inc., South San Francisco, California, USA
,
Amy BianCytokinetics, Inc., South San Francisco, California, USA
,
Jacqueline LeeCytokinetics, Inc., South San Francisco, California, USA
,
Donna BarraganCytokinetics, Inc., South San Francisco, California, USA
,
Jinsy A. AndrewsCytokinetics, Inc., South San Francisco, California, USA
& ; ON BEHALF OF THE BENEFIT-ALS STUDY GROUP show all
Pages 426-435 | Received 01 Oct 2015, Accepted 05 Dec 2015, Published online: 16 Mar 2016

References

  • Russell AJ, Hartman JJ, Hinken AC, et al. Activation of fast skeletal muscle troponin as a potential therapeutic approach for treating neuromuscular diseases. Nat Med. 2012;18:452–5.
  • Hansen R, Saikali KG, Chou W, et al. Tirasemtiv amplifies skeletal muscle response to nerve activation in humans. Muscle & Nerve. 2014:50:925–31.
  • Hwee DT, Kennedy A, Ryans J, et al. Fast skeletal muscle troponin activator tirasemtiv increases muscle function and performance in the B6SJL-SOD1-G93A ALS mouse model. PLoS One. 2014;9:e96921.
  • Shefner J, Cedarbaum JM, Cudkowicz ME, et al. Safety, tolerability and pharmacodynamics of a skeletal muscle activator in amyotrophic lateral sclerosis. Amyotroph Lateral Scler. 2012;13:430–8.
  • Shefner J, Wolff A, Meng L. The relationship between tirasemtiv serum concentration and functional outcomes in patients with ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2013;14:582–5.
  • Shefner JM, Watson ML, Meng L, Wolff AA, Team NCS. A study to evaluate safety and tolerability of repeated doses of tirasemtiv in patients with amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2013;14:574–81.
  • Brooks BR, Miller RG, Swash M, Munsat TL, World Federation of Neurology Research Group on Motor Neuron D. El Escorial revisited: revised criteria for the diagnosis of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord. 2000;1:293–9.
  • Cudkowicz M, van den Berg L, Shefner J, et al. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomized, double-blind, phase III trial. Lancet Neurol. 2013;11:1059–67.
  • Berry JD, Shefner JM, Conwit R, et al. Design and initial results of a multi-phase randomized trial of ceftriaxone in amyotrophic lateral sclerosis. PLoS One. 2013;8: e61177.
  • Dupuis L, Dengler R, Heneka MT, et al. A randomized, double-blind, placebo-controlled trial of pioglitazone in combination with riluzole in amyotrophic lateral sclerosis. PLoS One. 2012;7:e37885.
  • Lenglet T, Lacomblez L, Abitbol JL, et al. A phase II-III trial of olesoxime in subjects with amyotrophic lateral sclerosis. Eur J Neurol. 2014;21:529–36.
  • Group UK-LS, Morrison KE, Dhariwal S, et al. Lithium in patients with amyotrophic lateral sclerosis (LiCALS): a phase III multicentre, randomized, double-blind, placebo-controlled trial. Lancet Neurol. 2013;12:339–45.
  • Miller RG, Moore DH, Forshew DA, et al. Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis: examining a more efficient trial design. Neurology. 2011;77:973–9.
  • Aggarwal SP, Zinman L, Simpson E, et al. Safety and efficacy of lithium in combination with riluzole for treatment of amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled trial. Lancet Neurol. 2010;9:481–8.
  • Kaufmann P, Thompson JL, Levy G, et al. Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III. Ann Neurol. 2009;66:235–44.
  • Meininger V, Drory VE, Leigh PN, Ludolph A, Robberecht W, Silani V. Glatiramer acetate has no impact on disease progression in ALS at 40 mg/day: a double-blind, randomized, multicentre, placebo-controlled trial. Amyotroph Lateral Scler. 2009;10:378–83.
  • Sorenson EJ, Windbank AJ, Mandrekar JN, et al. Subcutaneous IGF-1 is not beneficial in two-year ALS trial. Neurology. 2008;71:1770–5.
  • Gordon PH, Moore DH, Miller RG, et al. Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomized trial. Lancet Neurol. 2007;6:1045–53.
  • Miller R, Bradley W, Cudkowicz M, et al. Phase II/III randomized trial of TCH346 in patients with ALS. Neurology. 2007;69:776–4.
  • Cederbaum JM, Stambler N, Malta E, et al. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessment of respiratory function. Journal of the Neurological Sciences.1999;169:13–21.
  • Cudkowicz ME, Shefner JM, Schoenfeld DA, et al. A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis. Neurology.2003;61:456–64.
  • Czaplinski A, Yen AA, Appel SH. Forced vital capacity (FVC) as an indicator of survival and disease progression in an ALS clinic population. J Neurol Neurosurg Psychiatry. 2006;77:390–2.
  • Traynor BJ, Zhang H, Shefner JM, et al. Functional outcome measures as clinical trial endpoints in ALS. Neurology.2004;63:1933–5.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Order Reprints Request Corporate Permissions

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

Request Academic Permissions

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.

Related research

People also read lists articles that other readers of this article have read.

Recommended articles lists articles that we recommend and is powered by our AI driven recommendation engine.

Cited by lists all citing articles based on Crossref citations.
Articles with the Crossref icon will open in a new tab.