Latest ALS Clinical Trial Information

Abe, K., Itoyama, Y., Sobue, G., Tsuji, S., Aoki, M., Doyu, M., Hamada, C., Kondo, K., Yoneoka, T., Akimoto, M., et al. (2014). Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients. Amyotroph Lateral Scler Frontotemporal Degener 15, 610-617. 10.3109/21678421.2014.959024.

Castrillo-Viguera, C., Grasso, D.L., Simpson, E., Shefner, J., and Cudkowicz, M.E. (2010). Clinical significance in the change of decline in ALSFRS-R. Amyotroph Lateral Scler 11, 178-180. 10.3109/17482960903093710.

Cudkowicz, M.E., Lindborg, S.R., Goyal, N.A., Miller, R.G., Burford, M.J., Berry, J.D., Nicholson, K.A., Mozaffar, T., Katz, J.S., Jenkins, L.J., et al. (2022). A randomized placebo-controlled phase 3 study of mesenchymal stem cells induced to secrete high levels of neurotrophic factors in amyotrophic lateral sclerosis. Muscle Nerve 65, 291-302. 10.1002/mus.27472.

Hayashi, N., Atsuta, N., Yokoi, D., Nakamura, R., Nakatochi, M., Katsuno, M., Izumi, Y., Kanai, K., Hattori, N., Taniguchi, A., et al. (2020). Prognosis of amyotrophic lateral sclerosis patients undergoing tracheostomy invasive ventilation therapy in Japan. J Neurol Neurosurg Psychiatry 91, 285-290. 10.1136/jnnp-2019-322213.

Hogg, M.C., Halang, L., Woods, I., Coughlan, K.S., and Prehn, J.H.M. (2018). Riluzole does not improve lifespan or motor function in three ALS mouse models. Amyotroph Lateral Scler Frontotemporal Degener 19, 438-445. 10.1080/21678421.2017.1407796.

Ishigaki, A., Aoki, M., Nagai, M., Warita, H., Kato, S., Kato, M., Nakamura, T., Funakoshi, H., and Itoyama, Y. (2007). Intrathecal delivery of hepatocyte growth factor from amyotrophic lateral sclerosis onset suppresses disease progression in rat amyotrophic lateral sclerosis model. J Neuropathol Exp Neurol 66, 1037-1044. 10.1097/nen.0b013e318159886b.

Ito, H., Wate, R., Zhang, J., Ohnishi, S., Kaneko, S., Ito, H., Nakano, S., and Kusaka, H. (2008). Treatment with edaravone, initiated at symptom onset, slows motor decline and decreases SOD1 deposition in ALS mice. Exp Neurol 213, 448-455. 10.1016/j.expneurol.2008.07.017.

Keiko Imamura, Yuishin Izumi, Makiko Nagai, Kazutoshi Nishiyama, Yasuhiro Watanabe, Ritsuko Hanajima, Naohiro Egawa, Takashi Ayaki, Ryosuke Oki, Koji Fujita, et al. (2022). Safety and tolerability of bosutinib in patients with amyotrophic lateral sclerosis (iDReAM study): a multicentre, open-label, dose-escalation phase 1 trial. eClinicalMedicine. 10.1016/j.eclinm.2022.101707.

Korobeynikov, V.A., Lyashchenko, A.K., Blanco-Redondo, B., Jafar-Nejad, P., and Shneider, N.A. (2022). Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nat Med 28, 104-116. 10.1038/s41591-021-01615-z.

Liu, E.Y., Russ, J., Cali, C.P., Phan, J.M., Amlie-Wolf, A., and Lee, E.B. (2019). Loss of Nuclear TDP-43 Is Associated with Decondensation of LINE Retrotransposons. Cell Rep 27, 1409-1421 e1406. 10.1016/j.celrep.2019.04.003.

Lobsiger, C.S., Boillee, S., Pozniak, C., Khan, A.M., McAlonis-Downes, M., Lewcock, J.W., and Cleveland, D.W. (2013). C1q induction and global complement pathway activation do not contribute to ALS toxicity in mutant SOD1 mice. Proc Natl Acad Sci U S A 110, E4385-4392. 10.1073/pnas.1318309110.

Miller, R.G., Mitchell, J.D., and Moore, D.H. (2012). Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Cochrane Database Syst Rev, CD001447. 10.1002/14651858.CD001447.pub3.

Miller, T.M., Cudkowicz, M.E., Genge, A., Shaw, P.J., Sobue, G., Bucelli, R.C., Chio, A., Van Damme, P., Ludolph, A.C., Glass, J.D., et al. (2022). Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med 387, 1099-1110. 10.1056/NEJMoa2204705.

Mitsubishi Tanabe Pharma America, I. (2022). Mitsubishi Tanabe Pharma America Presents Real-World Data on RADICAVA® (edaravone) at 2022 American Academy of Neurology Annual Meeting. https://www.prnewswire.com/news-releases/mitsubishi-tanabe-pharma-america-presents-real-world-data-on-radicava-edaravone-at-2022-american-academy-of-neurology-annual-meeting-301516649.html.

Mora, J.S., Bradley, W.G., Chaverri, D., Hernandez-Barral, M., Mascias, J., Gamez, J., Gargiulo-Monachelli, G.M., Moussy, A., Mansfield, C.D., Hermine, O., and Ludolph, A.C. (2021). Long-term survival analysis of masitinib in amyotrophic lateral sclerosis. Ther Adv Neurol Disord 14, 17562864211030365. 10.1177/17562864211030365.

Mora, J.S., Genge, A., Chio, A., Estol, C.J., Chaverri, D., Hernandez, M., Marin, S., Mascias, J., Rodriguez, G.E., Povedano, M., et al. (2020). Masitinib as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomized clinical trial. Amyotroph Lateral Scler Frontotemporal Degener 21, 5-14. 10.1080/21678421.2019.1632346.

Morimoto, S., Takahashi, S., Ito, D., Daté, Y., Okada, K., Chyi, C.M., Nishiyama, A., Suzuk, N., Fujimori, K., Takao, M., et al. (2022). Ropinirole hydrochloride for amyotrophic lateral sclerosis: A single-center, randomized feasibility, double-blind, placebo-controlled trial. medRxiv. https://doi.org/10.1101/2021.12.05.21267266.

Mueller, C., Berry, J.D., McKenna-Yasek, D.M., Gernoux, G., Owegi, M.A., Pothier, L.M., Douthwright, C.L., Gelevski, D., Luppino, S.D., Blackwood, M., et al. (2020). SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS. N Engl J Med 383, 151-158. 10.1056/NEJMoa2005056.

Oki, R., Izumi, Y., Fujita, K., Miyamoto, R., Nodera, H., Sato, Y., Sakaguchi, S., Nokihara, H., Kanai, K., Tsunemi, T., et al. (2022). Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. JAMA Neurol. 10.1001/jamaneurol.2022.0901.

Paganoni, S., Hendrix, S., Dickson, S.P., Knowlton, N., Berry, J.D., Elliott, M.A., Maiser, S., Karam, C., Caress, J.B., Owegi, M.A., et al. (2022). Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial. J Neurol Neurosurg Psychiatry. 10.1136/jnnp-2022-329024.

Paganoni, S., Macklin, E.A., Hendrix, S., Berry, J.D., Elliott, M.A., Maiser, S., Karam, C., Caress, J.B., Owegi, M.A., Quick, A., et al. (2020). Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med 383, 919-930. 10.1056/NEJMoa1916945.

Pioro, E.P., Brooks, B.R., Cummings, J., Schiffer, R., Thisted, R.A., Wynn, D., Hepner, A., Kaye, R., Safety, T., and Efficacy Results Trial of, A.V.P.i.P.B.A.I. (2010). Dextromethorphan plus ultra low-dose quinidine reduces pseudobulbar affect. Ann Neurol 68, 693-702. 10.1002/ana.22093.

Trias, E., Ibarburu, S., Barreto-Nunez, R., Babdor, J., Maciel, T.T., Guillo, M., Gros, L., Dubreuil, P., Diaz-Amarilla, P., Cassina, P., et al. (2016). Post-paralysis tyrosine kinase inhibition with masitinib abrogates neuroinflammation and slows disease progression in inherited amyotrophic lateral sclerosis. J Neuroinflammation 13, 177. 10.1186/s12974-016-0620-9.

Witzel, S., Maier, A., Steinbach, R., Grosskreutz, J., Koch, J.C., Sarikidi, A., Petri, S., Gunther, R., Wolf, J., Hermann, A., et al. (2022). Safety and Effectiveness of Long-term Intravenous Administration of Edaravone for Treatment of Patients With Amyotrophic Lateral Sclerosis. JAMA Neurol 79, 121-130. 10.1001/jamaneurol.2021.4893.

Woodruff, T.M., Costantini, K.J., Crane, J.W., Atkin, J.D., Monk, P.N., Taylor, S.M., and Noakes, P.G. (2008). The complement factor C5a contributes to pathology in a rat model of amyotrophic lateral sclerosis. J Immunol 181, 8727-8734. 10.4049/jimmunol.181.12.8727.

Writing-Group-Edaravone-ALS-Study-Group (2017). Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol 16, 505-512. 10.1016/S1474-4422(17)30115-1.

Xu, L., Liu, T., Liu, L., Yao, X., Chen, L., Fan, D., Zhan, S., and Wang, S. (2020). Global variation in prevalence and incidence of amyotrophic lateral sclerosis: a systematic review and meta-analysis. J Neurol 267, 944-953. 10.1007/s00415-019-09652-y.

Zhang, R., Bracci, P.M., Azhir, A., Forrest, B.D., and McGrath, M.S. (2022). Macrophage-Targeted Sodium Chlorite (NP001) Slows Progression of Amyotrophic Lateral Sclerosis (ALS) through Regulation of Microbial Translocation. Biomedicines 10. 10.3390/biomedicines10112907.

Zhou, B., Wang, H., Cai, Y., Wen, H., Wang, L., Zhu, M., Chen, Y., Yu, Y., Lu, X., Zhou, M., et al. (2020). FUS P525L mutation causing amyotrophic lateral sclerosis and movement disorders. Brain Behav 10, e01625. 10.1002/brb3.1625.