{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Kaufmann P"],"funding":["NCATS NIH HHS","NCRR NIH HHS","NINDS NIH HHS"],"pagination":["235-44"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC2854625"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["66(2)"],"pubmed_abstract":["<h4>Objective</h4>Amyotrophic lateral sclerosis (ALS) is a devastating, and currently incurable, neuromuscular disease in which oxidative stress and mitochondrial impairment are contributing to neuronal loss. Coenzyme Q10 (CoQ10), an antioxidant and mitochondrial cofactor, has shown promise in ALS transgenic mice, and in clinical trials for neurodegenerative diseases other than ALS. Our aims were to choose between two high doses of CoQ10 for ALS, and to determine if it merits testing in a Phase III clinical trial.<h4>Methods</h4>We designed and implemented a multicenter trial with an adaptive, two-stage, bias-adjusted, randomized, placebo-controlled, double-blind, Phase II design (n = 185). The primary outcome in both stages was a decline in the ALS Functional Rating Scale-revised (ALSFRSr"],"journal":["Annals of neurology"],"pubmed_title":["Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III."],"pmcid":["PMC2854625"],"funding_grant_id":["M01 RR001066","M01-RR023940","1 UL1 RR024156","UL1 TR000448","K12RR017648","K12 RR017648","M01-RR01066","R01 NS048555","R01 NS048125","UL1 RR024992","UL1 RR024156","R01 NS48125","M01 RR023940","R01 NS48555"],"pubmed_authors":["Mitsumoto H","Wulf C","Champion S","Rezania K","Herbelin L","Buchsbaum R","Goldstein JM","Clark C","Forshew D","Toenjes A","Scelsa S","DeBoo A","Pioro EP","Cutura D","Abrams B","Rowland LP","Tandan R","Scholtz D","Kittrell PP","Myers DA","Shefner J","Schierbecker JR","Fewell D","Krivickas LS","Katz J","McVey A","Kaufman P","Oskarsson B","Barsdorf AI","Heuer C","Caraganis A","Levy G","Butsch P","Potter CA","Eckenrode J","Watson ML","Conwit R","Armon C","Andrews J","Casey P","Pestronk A","Feldman S","Cudkowicz ME","DiMauro S","Novella SP","Taft J","Kelly K","Jones MC","Gleason R","Harrington G","Moxley K","English RA","Holloway R","Neville H","Swanson S","Soliven B","Kaufmann P","Rudnicki SA","Jackson CE","Gardner J","Barr C","Kasarskis EJ","Squire L","David WS","Battista V","Wells S","QALS Study Group","Hammack B","Villierme C","DiBernardo AB","Tiryaki E","Vecchio D","McDade S","Marra J","Gamage T","Manes S","Dick AR","Ringel SP","Vanderpool K","Wilkom G","Shrestha C","Traynor B","Taylor DG","Montes J","Chad D","Pulley D","Coffey C","Roos RP","Barsdorf A","Gordon PH","Reich E","Cumming J","Holford TR","Shaviers E","Grosso M","Sufit R","Conn S","Rollins Y","Marshall L","Barohn RJ","Kolb R","Levin B","Gutmann L","Hewitt B","Cordell J","Tierney A","Arbing R","Bednarz K","Miller RG","Florence JM","Walsh M","Heiman-Patterson T","Kruczek K","Malkus BC","Thompson JL","Lomen-Hoerth C"],"additional_accession":[]},"is_claimable":false,"name":"Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III.","description":"<h4>Objective</h4>Amyotrophic lateral sclerosis (ALS) is a devastating, and currently incurable, neuromuscular disease in which oxidative stress and mitochondrial impairment are contributing to neuronal loss. Coenzyme Q10 (CoQ10), an antioxidant and mitochondrial cofactor, has shown promise in ALS transgenic mice, and in clinical trials for neurodegenerative diseases other than ALS. Our aims were to choose between two high doses of CoQ10 for ALS, and to determine if it merits testing in a Phase III clinical trial.<h4>Methods</h4>We designed and implemented a multicenter trial with an adaptive, two-stage, bias-adjusted, randomized, placebo-controlled, double-blind, Phase II design (n = 185). The primary outcome in both stages was a decline in the ALS Functional Rating Scale-revised (ALSFRSr","dates":{"release":"2009-01-01T00:00:00Z","publication":"2009 Aug","modification":"2026-04-18T15:07:03.373Z","creation":"2026-04-07T14:40:38.898Z"},"accession":"S-EPMC2854625","cross_references":{"pubmed":["19743457"],"doi":["10.1002/ana.21743"]}}