A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)
Summary
The purpose of this study is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.
Timeline
- Start
- 2021-08-16
- Primary completion
- 2023-07-18
- Completion
- 2023-07-18
Outcome
Missed primary endpoint
Stopped: “The DMC recommended the trial be discontinued due to futility following a planned second interim analysis.”
paper The mean (SE) group difference in the ALSFRS-R score from baseline to week 24 was -1.1 (0.53; 95% CI, -2.17 to -0.08; P = .04, favoring placebo). PMID 40126464 ↗
paper CONCLUSIONS AND RELEVANCE: This randomized clinical trial failed to demonstrate efficacy for reldesemtiv in slowing functional decline in ALS. PMID 40126464 ↗
release “The DMC reviewed unblinded data from COURAGE-ALS and recommended the discontinuation of the clinical trial due to futility” cytokinetics.com ↗
release “As Previously Announced, Reldesemtiv Had No Effect on the Primary Endpoint of Change from Baseline in ALSFRS-R or Key Secondary Endpoints” cytokinetics.com ↗
Publications
- Rudnicki SA, Giacomelli E, Herder K, Ingre C, Kupfer S, Malik FI, Meng L, Paganoni S, Schellenberg K, Scirocco E, Simkins T, Wei J, Shefner JM; COURAGE-ALS Study Group. The Use of Durable Medical Equipment in COURAGE-ALS, a Phase 3, Multicentre, Randomized Clinical Trial for ALS. Muscle Nerve. 2026 Apr;73(4):608-614. doi: 10.1002/mus.70150. Epub 2026 Jan 19.
- Rudnicki SA, Gebrehiwet P, Kupfer S, Malik FI, Meng L, Simkins T, Wei J, Wolff AA, Shefner JM. Participant, site personnel and sponsor perspectives on decentralized trial features in COURAGE-ALS: a randomized clinical trial. Amyotroph Lateral Scler Frontotemporal Degener. 2025 Nov;26(7-8):812-820. doi: 10.1080/21678421.2025.2523941. Epub 2025 Jun 27.
- Rudnicki SA, Al-Chalabi A, Andrews JA, Chio A, Corcia P, Couratier P, Cudkowicz ME, De Carvalho M, Genge A, Hardiman O, Heiman-Patterson T, Henderson RD, Ingre C, Johnston W, Ludolph A, Maragakis NJ, Miller TM, Mora JS, Petri S, Simmons Z, Van Den Berg LH, Zinman L, Herder KE, Kupfer S, Malik FI, Meng L, Simkins TJ, Wei J, Wolff AA, Shefner JM; Courage-Als study group. Hospitalizations as an outcome measure in COURAGE-ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2025 Nov;26(7-8):802-811. doi: 10.1080/21678421.2025.2515907. Epub 2025 Jun 12.
- Shefner JM, Cudkowicz ME, Genge A, Hardiman O, Al-Chalabi A, Andrews JA, Chio A, Corcia P, Couratier P, de Carvalho M, Heiman-Patterson T, Henderson RD, Ingre C, Johnston W, Ludolph A, Maragakis NJ, Miller TM, Mora JS, Petri S, Simmons Z, van den Berg LH, Zinman L, Kupfer S, Malik FI, Meng L, Simkins TJ, Wei J, Wolff AA, Rudnicki SA; COURAGE-ALS Study Group. Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial. JAMA Neurol. 2025 May 1;82(5):477-485. doi: 10.1001/jamaneurol.2025.0241.
- Shefner JM, Al-Chalabi A, Andrews JA, Chio A, De Carvalho M, Cockroft BM, Corcia P, Couratier P, Cudkowicz ME, Genge A, Hardiman O, Heiman-Patterson T, Henderson RD, Ingre C, Jackson CE, Johnston W, Lechtzin N, Ludolph A, Maragakis NJ, Miller TM, Mora Pardina JS, Petri S, Simmons Z, Van Den Berg LH, Zinman L, Kupfer S, Malik FI, Meng L, Simkins TJ, Wei J, Wolff AA, Rudnicki SA. COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success. Amyotroph Lateral Scler Frontotemporal Degener. 2023 Aug;24(5-6):523-534. doi: 10.1080/21678421.2023.2216223. Epub 2023 May 30.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Reldesemtiv | Small molecule | 150 mg | Oral |
| Subject | Reldesemtiv | Small molecule | 300 mg | Oral |