TY  - JOUR
AU  - Shefner, Jeremy M
AU  - Cudkowicz, Merit E
AU  - Genge, Angela
AU  - Hardiman, Orla
AU  - Al-Chalabi, Ammar
AU  - Andrews, Jinsy A
AU  - Chio, Adriano
AU  - Corcia, Philippe
AU  - Couratier, Philippe
AU  - de Carvalho, Mamede
AU  - Heiman-Patterson, Terry
AU  - Henderson, Robert D
AU  - Ingre, Caroline
AU  - Johnston, Wendy
AU  - Ludolph, Albert
AU  - Maragakis, Nicholas J
AU  - Miller, Timothy M
AU  - Mora, Jesus S
AU  - Petri, Susanne
AU  - Simmons, Zachary
AU  - van den Berg, Leonard H
AU  - Zinman, Lorne
AU  - Kupfer, Stuart
AU  - Malik, Fady I
AU  - Meng, Lisa
AU  - Simkins, Tyrell J
AU  - Wei, Jenny
AU  - Wolff, Andrew A
AU  - Rudnicki, Stacy A
TI  - Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.
JO  - JAMA neurology
VL  - 82
IS  - 5
SN  - 2168-6149
CY  - Chicago, Ill.
PB  - American Medical Association
M1  - DZNE-2025-00603
SP  - 477 - 485
PY  - 2025
AB  - 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
KW  - Humans
KW  - Amyotrophic Lateral Sclerosis: drug therapy
KW  - Male
KW  - Female
KW  - Middle Aged
KW  - Double-Blind Method
KW  - Aged
KW  - Treatment Outcome
KW  - Oxadiazoles: therapeutic use
KW  - Disease Progression
KW  - Pyrazines
KW  - Pyrimidines
KW  - Imidazoles
KW  - Pyrroles
KW  - Pyridines
KW  - CK-2017357 (NLM Chemicals)
KW  - Oxadiazoles (NLM Chemicals)
KW  - reldesemtiv (NLM Chemicals)
KW  - Pyrazines (NLM Chemicals)
KW  - Pyrimidines (NLM Chemicals)
KW  - Imidazoles (NLM Chemicals)
KW  - Pyrroles (NLM Chemicals)
KW  - Pyridines (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:40126464
C2  - pmc:PMC11933997
DO  - DOI:10.1001/jamaneurol.2025.0241
UR  - https://pub.dzne.de/record/278570
ER  -