Clinical trial NCT04244630 - References

Amyotrophic Lateral Sclerosis (ALS) — Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial

Citation(s)

•   Akao Y, Maruyama W, Shimizu S, Yi H, Nakagawa Y, Shamoto-Nagai M, Youdim MB, Tsujimoto Y, Naoi M
  Mitochondrial permeability transition mediates apoptosis induced by N-methyl(R)salsolinol, an endogenous neurotoxin, and is inhibited by Bcl-2 and rasagiline, N-propargyl-1(R)-aminoindan. J Neurochem. 2002 Aug;82(4):913-23.
•   Benatar M, Wuu J, Andersen PM, Lombardi V, Malaspina A
  Neurofilament light: A candidate biomarker of presymptomatic amyotrophic lateral sclerosis and phenoconversion. Ann Neurol. 2018 Jul;84(1):130-139. doi: 10.1002/ana.25276. Epub 2018 Aug 16.
•   Brooks BR, Miller RG, Swash M, Munsat TL; World Federation of Neurology Research Group on Motor Neuron Diseases
  El Escorial revisited: revised criteria for the diagnosis of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord. 2000 Dec;1(5):293-9. Review.
•   Brooks BR
  El Escorial World Federation of Neurology criteria for the diagnosis of amyotrophic lateral sclerosis. Subcommittee on Motor Neuron Diseases/Amyotrophic Lateral Sclerosis of the World Federation of Neurology Research Group on Neuromuscular Diseases and the El Escorial "Clinical limits of amyotrophic lateral sclerosis" workshop contributors. J Neurol Sci. 1994 Jul;124 Suppl:96-107.
•   Cruz MP
  Edaravone (Radicava): A Novel Neuroprotective Agent for the Treatment of Amyotrophic Lateral Sclerosis. P T. 2018 Jan;43(1):25-28.
•   Cudkowicz M, Bozik ME, Ingersoll EW, Miller R, Mitsumoto H, Shefner J, Moore DH, Schoenfeld D, Mather JL, Archibald D, Sullivan M, Amburgey C, Moritz J, Gribkoff VK
  The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis. Nat Med. 2011 Nov 20;17(12):1652-6. doi: 10.1038/nm.2579.
•   de la Rubia JE, Drehmer E, Platero JL, Benlloch M, Caplliure-Llopis J, Villaron-Casales C, de Bernardo N, AlarcÓn J, Fuente C, Carrera S, Sancho D, GarcÍa-Pardo P, Pascual R, JuÁrez M, Cuerda-Ballester M, Forner A, Sancho-Castillo S, Barrios C, Obrador E, Marchio P, Salvador R, Holmes HE, Dellinger RW, Guarente L, Estrela JM
  Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Feb;20(1-2):115-122. doi: 10.1080/21678421.2018.1536152. Epub 2019 Jan 22.
•   Desai VG, Herman EH, Moland CL, Branham WS, Lewis SM, Davis KJ, George NI, Lee T, Kerr S, Fuscoe JC
  Development of doxorubicin-induced chronic cardiotoxicity in the B6C3F1 mouse model. Toxicol Appl Pharmacol. 2013 Jan 1;266(1):109-21. doi: 10.1016/j.taap.2012.10.025. Epub 2012 Nov 7.
•   Doble A
  The pharmacology and mechanism of action of riluzole. Neurology. 1996 Dec;47(6 Suppl 4):S233-41. Review.
•   Eisen A, Schulzer M, MacNeil M, Pant B, Mak E
  Duration of amyotrophic lateral sclerosis is age dependent. Muscle Nerve. 1993 Jan;16(1):27-32.
•   Evers BM, Rodriguez-Navas C, Tesla RJ, Prange-Kiel J, Wasser CR, Yoo KS, McDonald J, Cenik B, Ravenscroft TA, Plattner F, Rademakers R, Yu G, White CL 3rd, Herz J
  Lipidomic and Transcriptomic Basis of Lysosomal Dysfunction in Progranulin Deficiency. Cell Rep. 2017 Sep 12;20(11):2565-2574. doi: 10.1016/j.celrep.2017.08.056.
•   Feneberg E, Oeckl P, Steinacker P, Verde F, Barro C, Van Damme P, Gray E, Grosskreutz J, Jardel C, Kuhle J, Koerner S, Lamari F, Amador MDM, Mayer B, Morelli C, Muckova P, Petri S, Poesen K, Raaphorst J, Salachas F, Silani V, Stubendorff B, Turner MR, Verbeek MM, Weishaupt JH, Weydt P, Ludolph AC, Otto M
  Multicenter evaluation of neurofilaments in early symptom onset amyotrophic lateral sclerosis. Neurology. 2018 Jan 2;90(1):e22-e30. doi: 10.1212/WNL.0000000000004761. Epub 2017 Dec 6.
•   Gaiottino J, Norgren N, Dobson R, Topping J, Nissim A, Malaspina A, Bestwick JP, Monsch AU, Regeniter A, Lindberg RL, Kappos L, Leppert D, Petzold A, Giovannoni G, Kuhle J
  Increased neurofilament light chain blood levels in neurodegenerative neurological diseases. PLoS One. 2013 Sep 20;8(9):e75091. doi: 10.1371/journal.pone.0075091. eCollection 2013.
•   Gubbay SS, Kahana E, Zilber N, Cooper G, Pintov S, Leibowitz Y
  Amyotrophic lateral sclerosis. A study of its presentation and prognosis. J Neurol. 1985;232(5):295-300.
•   Hokkanen SRK, Hunter S, Polvikoski TM, Keage HAD, Minett T, Matthews FE, Brayne C; MRC CFAS and CC75C Study Group
  Hippocampal sclerosis, hippocampal neuron loss patterns and TDP-43 in the aged population. Brain Pathol. 2018 Jul;28(4):548-559. doi: 10.1111/bpa.12556. Epub 2017 Sep 25.
•   Huisman MH, de Jong SW, van Doormaal PT, Weinreich SS, Schelhaas HJ, van der Kooi AJ, de Visser M, Veldink JH, van den Berg LH
  Population based epidemiology of amyotrophic lateral sclerosis using capture-recapture methodology. J Neurol Neurosurg Psychiatry. 2011 Oct;82(10):1165-70. doi: 10.1136/jnnp.2011.244939. Epub 2011 May 27.
•   Kaufmann P, Levy G, Montes J, Buchsbaum R, Barsdorf AI, Battista V, Arbing R, Gordon PH, Mitsumoto H, Levin B, Thompson JL; QALS study group
  Excellent inter-rater, intra-rater, and telephone-administered reliability of the ALSFRS-R in a multicenter clinical trial. Amyotroph Lateral Scler. 2007 Feb;8(1):42-6.
•   Keage HA, Hunter S, Matthews FE, Ince PG, Hodges J, Hokkanen SR, Highley JR, Dening T, Brayne C
  TDP-43 pathology in the population: prevalence and associations with dementia and age. J Alzheimers Dis. 2014;42(2):641-50. doi: 10.3233/JAD-132351.
•   Logroscino G, Traynor BJ, Hardiman O, Chiò A, Mitchell D, Swingler RJ, Millul A, Benn E, Beghi E; EURALS
  Incidence of amyotrophic lateral sclerosis in Europe. J Neurol Neurosurg Psychiatry. 2010 Apr;81(4):385-90. doi: 10.1136/jnnp.2009.183525. Epub 2009 Aug 25.
•   Lu CH, Macdonald-Wallis C, Gray E, Pearce N, Petzold A, Norgren N, Giovannoni G, Fratta P, Sidle K, Fish M, Orrell R, Howard R, Talbot K, Greensmith L, Kuhle J, Turner MR, Malaspina A
  Neurofilament light chain: A prognostic biomarker in amyotrophic lateral sclerosis. Neurology. 2015 Jun 2;84(22):2247-57. doi: 10.1212/WNL.0000000000001642. Epub 2015 May 1. Erratum in: Neurology. 2015 Sep 8;85(10):921.
•   Neumann M, Sampathu DM, Kwong LK, Truax AC, Micsenyi MC, Chou TT, Bruce J, Schuck T, Grossman M, Clark CM, McCluskey LF, Miller BL, Masliah E, Mackenzie IR, Feldman H, Feiden W, Kretzschmar HA, Trojanowski JQ, Lee VM
  Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis. Science. 2006 Oct 6;314(5796):130-3.
•   O'Toole O, Traynor BJ, Brennan P, Sheehan C, Frost E, Corr B, Hardiman O
  Epidemiology and clinical features of amyotrophic lateral sclerosis in Ireland between 1995 and 2004. J Neurol Neurosurg Psychiatry. 2008 Jan;79(1):30-2. Epub 2007 Jul 18.
•   Pieper M, Scheffold C, Duwe S, Rossig C, Bisping G, Stelljes M, Tedder TF, Jurgens H, Berdel WE, Kienast J
  Immunotherapy of B-cell malignancies with genetically engineered human CD8+ natural killer T cells. Leukemia. 2006 Apr;20(4):729-32.
•   Radford RA, Morsch M, Rayner SL, Cole NJ, Pountney DL, Chung RS
  The established and emerging roles of astrocytes and microglia in amyotrophic lateral sclerosis and frontotemporal dementia. Front Cell Neurosci. 2015 Oct 27;9:414. doi: 10.3389/fncel.2015.00414. eCollection 2015. Review.
•   Rowland LP, Shneider NA
  Amyotrophic lateral sclerosis. N Engl J Med. 2001 May 31;344(22):1688-700. Review.
•   Taylor JP, Brown RH Jr, Cleveland DW
  Decoding ALS: from genes to mechanism. Nature. 2016 Nov 10;539(7628):197-206. doi: 10.1038/nature20413. Review.
•   Thouvenot E, Demattei C, Lehmann S, Maceski-Maleska A, Hirtz C, Juntas-Morales R, Pageot N, Esselin F, Alphandéry S, Vincent T, Camu W
  Serum neurofilament light chain at time of diagnosis is an independent prognostic factor of survival in amyotrophic lateral sclerosis. Eur J Neurol. 2020 Feb;27(2):251-257. doi: 10.1111/ene.14063. Epub 2019 Sep 18.
•   van Es MA, Hardiman O, Chio A, Al-Chalabi A, Pasterkamp RJ, Veldink JH, van den Berg LH
  Amyotrophic lateral sclerosis. Lancet. 2017 Nov 4;390(10107):2084-2098. doi: 10.1016/S0140-6736(17)31287-4. Epub 2017 May 25. Review.
•   Verde F, Steinacker P, Weishaupt JH, Kassubek J, Oeckl P, Halbgebauer S, Tumani H, von Arnim CAF, Dorst J, Feneberg E, Mayer B, Müller HP, Gorges M, Rosenbohm A, Volk AE, Silani V, Ludolph AC, Otto M
  Neurofilament light chain in serum for the diagnosis of amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry. 2019 Feb;90(2):157-164. doi: 10.1136/jnnp-2018-318704. Epub 2018 Oct 11.
•   Wang G, Han T, Nijhawan D, Theodoropoulos P, Naidoo J, Yadavalli S, Mirzaei H, Pieper AA, Ready JM, McKnight SL
  P7C3 neuroprotective chemicals function by activating the rate-limiting enzyme in NAD salvage. Cell. 2014 Sep 11;158(6):1324-1334. doi: 10.1016/j.cell.2014.07.040.
•   Wittie M, Nelson LM, Usher S, Ward K, Benatar M
  Utility of capture-recapture methodology to assess completeness of amyotrophic lateral sclerosis case ascertainment. Neuroepidemiology. 2013;40(2):133-41. doi: 10.1159/000342156. Epub 2012 Oct 24.
•   Yin TC, Britt JK, De Jesús-Cortés H, Lu Y, Genova RM, Khan MZ, Voorhees JR, Shao J, Katzman AC, Huntington PJ, Wassink C, McDaniel L, Newell EA, Dutca LM, Naidoo J, Cui H, Bassuk AG, Harper MM, McKnight SL, Ready JM, Pieper AA
  P7C3 neuroprotective chemicals block axonal degeneration and preserve function after traumatic brain injury. Cell Rep. 2014 Sep 25;8(6):1731-1740. doi: 10.1016/j.celrep.2014.08.030. Epub 2014 Sep 15.

Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial

Details for clinical trial NCT04244630