%0 Journal Article
%A Shefner, Jeremy M
%A Cudkowicz, Merit E
%A Genge, Angela
%A Hardiman, Orla
%A Al-Chalabi, Ammar
%A Andrews, Jinsy A
%A Chio, Adriano
%A Corcia, Philippe
%A Couratier, Philippe
%A de Carvalho, Mamede
%A Heiman-Patterson, Terry
%A Henderson, Robert D
%A Ingre, Caroline
%A Johnston, Wendy
%A Ludolph, Albert
%A Maragakis, Nicholas J
%A Miller, Timothy M
%A Mora, Jesus S
%A Petri, Susanne
%A Simmons, Zachary
%A van den Berg, Leonard H
%A Zinman, Lorne
%A Kupfer, Stuart
%A Malik, Fady I
%A Meng, Lisa
%A Simkins, Tyrell J
%A Wei, Jenny
%A Wolff, Andrew A
%A Rudnicki, Stacy A
%T Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.
%J JAMA neurology
%V 82
%N 5
%@ 2168-6149
%C Chicago, Ill.
%I American Medical Association
%M DZNE-2025-00603
%P 477 - 485
%D 2025
%X 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.To assess the effect of reldesemtiv vs placebo on functional outcomes in ALS.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 first for futility and the second for futility and possible resizing. This was a hybrid decentralized trial with approximately half the trial visits performed remotely and the remaining visits in the clinic. Eligible participants met criteria for definite, probable, or possible ALS with lower motor neuron signs by modified El Escorial Criteria, ALS symptoms for 24 months or less, ALS Functional Rating Scale-Revised (ALSFRS-R) total score of 44 or less, and forced vital capacity of greater than or equal to 65
%K Humans
%K Amyotrophic Lateral Sclerosis: drug therapy
%K Male
%K Female
%K Middle Aged
%K Double-Blind Method
%K Aged
%K Treatment Outcome
%K Oxadiazoles: therapeutic use
%K Disease Progression
%K Pyrazines
%K Pyrimidines
%K Imidazoles
%K Pyrroles
%K Pyridines
%K CK-2017357 (NLM Chemicals)
%K Oxadiazoles (NLM Chemicals)
%K reldesemtiv (NLM Chemicals)
%K Pyrazines (NLM Chemicals)
%K Pyrimidines (NLM Chemicals)
%K Imidazoles (NLM Chemicals)
%K Pyrroles (NLM Chemicals)
%K Pyridines (NLM Chemicals)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:40126464
%2 pmc:PMC11933997
%R 10.1001/jamaneurol.2025.0241
%U https://pub.dzne.de/record/278570