COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success

Author:

Shefner Jeremy M.1ORCID,Al-Chalabi Ammar2ORCID,Andrews Jinsy A.3ORCID,Chio Adriano4ORCID,De Carvalho Mamede5ORCID,Cockroft Bettina M.6,Corcia Philippe7ORCID,Couratier Philippe8ORCID,Cudkowicz Merit E.9ORCID,Genge Angela10ORCID,Hardiman Orla11ORCID,Heiman-Patterson Terry12ORCID,Henderson Robert D.13ORCID,Ingre Caroline14ORCID,Jackson Carlayne E.15ORCID,Johnston Wendy16,Lechtzin Noah17ORCID,Ludolph Albert18ORCID,Maragakis Nicholas J.19ORCID,Miller Timothy M.20ORCID,Mora Pardina Jesus S.21ORCID,Petri Susanne22ORCID,Simmons Zachary23ORCID,Van Den Berg Leonard H.24ORCID,Zinman Lorne25ORCID,Kupfer Stuart26ORCID,Malik Fady I.26ORCID,Meng Lisa26,Simkins Tyrell J.26ORCID,Wei Jenny26,Wolff Andrew A.26ORCID,Rudnicki Stacy A.26ORCID

Affiliation:

1. Barrow Neurological Institute, University of Arizona, and Creighton University, Phoenix, AZ, USA

2. King’s College London, London, UK

3. The Neurological Institute of New York, Columbia University Irving Medical Center, New York, NY, USA

4. Rita Levi Montalcini’ Department of Neuroscience, University of Turin, Turin, Italy

5. Department of Neurosciences of Centro Hospitalar Universitário Lisboa-Norte, Faculty of Medicine, Centro de Estudos Egas Moniz, Institute of Molecular Medicine, Universidade de Lisboa, Lisbon, Portugal

6. Sangamo Therapeutics, Inc, Brisbane, CA, USA

7. Centre de Réference SLA, CHU Bretonneau, Tours, France

8. Neurology Department, LS Centre CHU Dupuytren, Limoges, France

9. Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, MA, USA

10. Montreal Neurological Institute, Montreal, QC, Canada

11. Academic Unit of Neurology, Trinity Biomedical Sciences Institute, Trinity College, Dublin, Ireland

12. Neurology Lewis Katz School of Medicine at Temple University, Philadelphia, PA, USA

13. UQ Centre for Clinical Research, The University of Queensland, Brisbane, Australia

14. Department of Clinical Neuroscience, Karolinska Institute, Stockholm, Sweden

15. University of Texas Health Science Center at San Antonio, San Antonio, TX, USA

16. University of Alberta, Edmonton, AB, Canada

17. Johns Hopkins University School of Medicine, Baltimore, MD, USA

18. UULM Clinic of Neurology, Ulm University, Ulm, Germany

19. Johns Hopkins ALS Clinical Trials Unit, Johns Hopkins University, Baltimore, MD, USA

20. Washington University School of Medicine, St. Louis, MO, USA

21. ALS Unit, Hospital Universitario San Rafael, Madrid, Spain

22. Department of Neurology, Hannover Medical School, Hannover, Germany

23. Penn State Health Milton S Hershey Medical Center, Hershey, PA, USA

24. Department of Neurology, UMC Utrecht Brain Center, Netherlands ALS Centre, University Medical Center Utrecht, Utrecht, Netherlands

25. Sunnybrook Health Sciences Centre, Toronto, ON, Canada, and

26. Cytokinetics, Incorporated, South San Francisco, CA, USA

Funder

Cytokinetics

Publisher

Informa UK Limited

Subject

Neurology (clinical),Neurology

Reference20 articles.

1. Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients

2. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial

3. Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

4. Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosis

5. US Department of Health and Human Services US Food and Drug Administration Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER). Amyotrophic lateral sclerosis: developing drugs for treatment. Guidance for industry. https://www.fda.gov/media/130964/download. Accessed August 3 2022.

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