Prescription and acceptance of durable medical equipment in FORTITUDE-ALS, a study of reldesemtiv in ALS: post hoc analyses of a randomized, double-blind, placebo-controlled clinical trial

Abstract

Objective: To evaluate the possible effect of reldesemtiv, a fast skeletal muscle troponin activator, on prescription and acceptance of durable medical equipment (DME) in the FORTITUDE-ALS trial. Methods: Health economic outcome information was collected in FORTITUDE-ALS (NCT03160898); sites recorded if and when DME, specifically manual or power wheelchairs, gastrostomy tubes, noninvasive ventilators, or augmentative language devices, was prescribed by a physician and accepted by the patient (DME-PAP) during the trial. Acceptance was defined as the patient agreeing the item was needed. Cox regression analysis compared time to DME-PAP for each reldesemtiv dose with placebo. Post hoc analyses evaluated all reldesemtiv doses compared with placebo. Results: At least one DME item was prescribed and accepted by 33/114 (28.9%) of placebo patients, 19/112 (17.0%) of patients receiving reldesemtiv 150 mg bid, 24/113 (21.2%) receiving 300 mg bid, and 29/117 (24.8%) receiving 450 mg bid. The proportion of new DME-PAP was significantly lower in patients receiving reldesemtiv 150 mg bid vs placebo (17.0% vs 28.9%, p = 0.032). The hazard ratio versus placebo for accepting at least one DME item for all reldesemtiv doses combined was 0.61 (confidence interval: 0.39, 0.96, p = 0.032). 25% of placebo patients were prescribed and agreed to obtain a DME item by 84 days; this threshold was met for reldesemtiv-treated patients at 120 days. Conclusions: Results suggest ALS patients receiving reldesemtiv may have lower risk of and delayed need for DME related to impaired mobility, breathing, swallowing, or speaking; this delay is consistent with other measures indicating delay in disease progression.

Keywords:

• Disease burden
• clinical trial
• reldesemtiv
• FORTITUDE-ALS
• health outcomes research

Acknowledgements

We wish to thank the participants of FORTITUDE-ALS and their families for their contributions to this clinical trial, the investigators of FORTITUDE-ALS, and members of the Data Monitoring Committee and Steering Committee.

Declaration of interest

JAA has served as consultant for AL-S Pharma, Avexis, Biogen, Cytokinetics, Denali and Wave Live Sciences; has received research support from Biogen, Novartis, Orion and NIH; and is a former employee of Cytokinetics.

AG has served as a consultant for AB Sciences, AL-S Pharma, Avexis, Biogen, Cytokinetics, Incorporated, MT Pharma America, and Roche.

CJ has served as a consultant for Argenex, Cytokinetics, Incorporated, ITF Pharma, MT Pharma America, and Strongbridge Pharmaceuticals; served on Speaker’s Bureau for Avanir, CSL Behring, MT Pharma America, and Strongbridge Pharmaceuticals; has received research support from Amylyx, Cytokinetics, Incorporated, and NIH; and is currently serving as a member of a Data Safety Monitoring Board for Anelixis, Brainstorm, and Mallinckrodt.

NL has served as a consultant/advisor for Cytokinetics, Incorporated, Hill-Rom, and Vertex; and has received research support from AstraZeneca and Vertex.

TMM has served as a consultant/advisor for Biogen and Cytokinetics, Incorporated; has received research support from Biogen and Ionis; and receives licensing fees from C2N.

JMS has served as a consultant for Biogen, Biohaven, Cytokinetics, Incorporated, MT Pharma America, and Novartis; has received research support from Amylyx, Biogen, Biohaven, Biotie, Cytokinetics, Incorporated, MT Pharma America, Neuraltus, and Orphazyme; and has received compensation from UpToDate for serving as neuromuscular section editor.

BMC was an employee of Cytokinetics, Incorporated at the time of the study and owns stock in Cytokinetics, Incorporated. SAR, FIM, LM, JW, and AAW are employees of and own stock in Cytokinetics, Incorporated.

Data availability statement

Data reported herein are part of a sponsor-led clinical development program that is ongoing, and thus complete datasets for the trial will not be made available with this report.

Additional information

Funding

The FORTITUDE-ALS trial was conducted by Cytokinetics, Incorporated, South San Francisco, CA, USA and funded by Astellas Pharma Inc., Tokyo, Japan, as part of the collaboration between Cytokinetics, Incorporated and Astellas Pharma Inc.