biostudies-literatureUnknown82(5)Shefner JM<h4>Importance</h4>Treatment options for amyotrophic lateral sclerosis (ALS) remain suboptimal. Results from a phase 2 study of reldesemtiv in ALS suggested that it may slow disease progression.<h4>Objective</h4>To assess the effect of reldesemtiv vs placebo on functional outcomes in ALS.<h4>Design, setting, and participants</h4>A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (COURAGE-ALS) was a double-blind, placebo-controlled phase 3 randomized clinical trial conducted at 83 ALS centers in 16 countries from August 2021 to July 2023. The first 24-week period was placebo controlled vs reldesemtiv. All participants received reldesemtiv during the second 24-week period with a 4-week follow-up. Two interim analyses were planned, the fiJAMA neurology477-485https://www.ebi.ac.uk/biostudies/studies/S-EPMC11933997biostudies-literatureReldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.PMC11933997Johnston Wvan den Berg LHQuan DCreigh PYoung CFee DMaragakis NJStatland JGrogan JZwicker JRezania KCOURAGE-ALS Study GroupCouratier PKusma-Kozakiewicz MGoyal NVucic SShoesmith CSo YMeyer TGroßkreutz JRodiger ASimkins TJKiernan MCChahin NShefner JMKuenzler RZinman LGwathmey KVazquez-Costa JFTandan RSchultz DPavlakis PSoriani MHBodkin CLudolph ABayat ETurnbull JLewis RHardiman OKatz JMiller TMSalachas FGoutman SOwegi MAndrews JADionne ARothstein JCalvo ASimmons ZKupfer SAttarian SLunetta CPatee GMccluskey LHPulley MMora JSMatte GSwenson ALadha SBradshaw DPetri SOskarrson BSalveson LNilsson CCudkowicz MEPovedano MSilani VNeedham MCerri FWei JGenge APaganoni SWeber MHenderson RDIngre CWeydt PDanel VOlney NRudnicki SACorcia PSchellenberg Kde Carvalho MHeiman-Patterson TO'Connell CMMalik FIArcilla-London XChio AAl-Chalabi APeltier AMeng LBernard EVu TWolff AAKorngut LHeitzman DfalseReldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.<h4>Importance</h4>Treatment options for amyotrophic lateral sclerosis (ALS) remain suboptimal. Results from a phase 2 study of reldesemtiv in ALS suggested that it may slow disease progression.<h4>Objective</h4>To assess the effect of reldesemtiv vs placebo on functional outcomes in ALS.<h4>Design, setting, and participants</h4>A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (COURAGE-ALS) was a double-blind, placebo-controlled phase 3 randomized clinical trial conducted at 83 ALS centers in 16 countries from August 2021 to July 2023. The first 24-week period was placebo controlled vs reldesemtiv. All participants received reldesemtiv during the second 24-week period with a 4-week follow-up. Two interim analyses were planned, the fi2025-01-01T00:00:00Z2025 May2026-06-01T10:16:09.289Z2025-07-13T03:04:54.398ZS-EPMC119339974012646410.1001/jamaneurol.2025.0241