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(2022)
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
(2020)
Trial of sodium phenylbutyrate-taurursodiol for amyotrophic lateral sclerosis, 383
S. Pinto, M. Carvalho (2019)
SVC Is a Marker of Respiratory Decline Function, Similar to FVC, in Patients With ALSFrontiers in Neurology, 10
(2022)
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
P. Quanjer, S. Stanojevic, T. Cole, X. Baur, G. Hall, B. Culver, P. Enright, J. Hankinson, M. Ip, Jinping Zheng, J. Stocks (2012)
Multi-ethnic reference values for spirometry for the 3–95-yr age range: the global lung function 2012 equationsEuropean Respiratory Journal, 40
(2017)
Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial, 16
Jinsy Andrews, T. Miller, Vipin Vijayakumar, R. Stoltz, Joyce James, L. Meng, A. Wolff, F. Malik (2017)
CK‐2127107 amplifies skeletal muscle response to nerve activation in humansMuscle & Nerve, 57
C. Mehta, S. Pocock (2011)
Adaptive increase in sample size when interim results are promising: A practical guide with examplesStatistics in Medicine, 30
S. Pinto, M. Carvalho (2017)
Correlation between Forced Vital Capacity and Slow Vital Capacity for the assessment of respiratory involvement in Amyotrophic Lateral Sclerosis: a prospective studyAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 18
R. Eijk, J. Bakers, M. Es, M. Eijkemans, L. Berg (2019)
Implications of spirometric reference values for amyotrophic lateral sclerosisAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 20
B. Johnsen, K. Pugdahl, A. Fuglsang-Frederiksen, K. Kollewe, L. Paracka, Reinhard Dengler, J. Camdessanche, W. Nix, R. Liguori, I. Schofield, L. Maderna, D. Czell, C. Neuwirth, Markus Weber, V. Drory, A. Abraham, Michael Swash, M. Carvalho (2019)
Diagnostic criteria for amyotrophic lateral sclerosis: A multicentre study of inter-rater variation and sensitivityClinical Neurophysiology, 130
(1965)
Diagnostic criteriaActa Neurologica Scandinavica, 41
J. Shefner, Jinsy Andrews, A. Genge, C. Jackson, N. Lechtzin, T. Miller, Bettina Cockroft, L. Meng, Jenny Wei, A. Wolff, F. Malik, C. Bodkin, B. Brooks, J. Caress, A. Dionne, D. Fee, Stephen Goutman, N. Goyal, O. Hardiman, G. Hayat, T. Heiman-Patterson, Daragh Heitzman, R. Henderson, W. Johnston, C. Karam, M. Kiernan, S. Kolb, L. Korngut, S. Ladha, G. Matte, J. Mora, M. Needham, B. Oskarsson, Gary Pattee, E. Pioro, M. Pulley, D. Quan, K. Rezania, K. Schellenberg, D. Schultz, C. Shoesmith, Z. Simmons, J. Statland, S. Sultan, A. Swenson, L. Berg, T. Vu, S. Vucic, M. Weiss, Ashley Whyte-Rayson, J. Wymer, L. Zinman, S. Rudnicki (2020)
A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALSAmyotrophic lateral sclerosis & frontotemporal degeneration, 22
Y. Chen, D. DeMets, K. Lan (2004)
Increasing the sample size when the unblinded interim result is promisingStatistics in Medicine, 23
S. Paganoni, S. Hendrix, S. Dickson, N. Knowlton, E. Macklin, J. Berry, Michael Elliott, S. Maiser, C. Karam, J. Caress, M. Owegi, Adam Quick, J. Wymer, Stephen Goutman, Daragh Heitzman, T. Heiman-Patterson, C. Jackson, C. Quinn, J. Rothstein, E. Kasarskis, J. Katz, Liberty Jenkins, S. Ladha, T. Miller, S. Scelsa, T. Vu, Christina Fournier, J. Glass, Kristina Johnson, A. Swenson, N. Goyal, Gary Pattee, P. Andres, Suma Babu, Marianne Chase, D. Dagostino, Meghan Hall, Gale Kittle, Matthew Eydinov, Michelle McGovern, Joseph Ostrow, L. Pothier, Rebecca Randall, J. Shefner, A. Sherman, M. Pierre, E. Tustison, P. Vigneswaran, Jason Walker, Hong Yu, James Chan, J. Wittes, Zi‐Fan Yu, J. Cohen, J. Klee, K. Leslie, R. Tanzi, Walter Gilbert, P. Yeramian, D. Schoenfeld, M. Cudkowicz (2020)
Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosisMuscle & Nerve, 63
The PHOENIX trial: phase III trial of AMX0035 for amyotrophic lateral sclerosis treatment
P. Gao, J. Ware, C. Mehta (2008)
Sample Size Re-Estimation for Adaptive Sequential Design in Clinical TrialsJournal of Biopharmaceutical Statistics, 18
K. Abe, Y. Itoyama, G. Sobue, S. Tsuji, M. Aoki, M. Doyu, C. Hamada, K. Kondo, Takatomo Yoneoka, M. Akimoto, H. Yoshino (2014)
Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patientsAmyotrophic Lateral Sclerosis & Frontotemporal Degeneration, 15
(2017)
MCI-186) ALS 19 Study Group Writing Group. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial
The HEALEY ALS platform trial
Adriaan Jongh, Nathalie Braun, Markus Weber, M. Es, Pegah Masrori, J. Veldink, P. Damme, L. Berg, R. Eijk (2022)
Characterising ALS disease progression according to El Escorial and Gold Coast criteriaJournal of Neurology, Neurosurgery, and Psychiatry, 93
B. Brooks, Robert Miller, M. Swash, T. Munsat (2000)
El Escorial revisited: Revised criteria for the diagnosis of amyotrophic lateral sclerosisAmyotrophic Lateral Sclerosis and Other Motor Neuron Disorders, 1
P. Gebrehiwet, L. Meng, S. Rudnicki, P. Sarocco, Jenny Wei, A. Wolff, A. Chiò, Jinsy Andrews, A. Genge, C. Jackson, N. Lechtzin, Timothy Miller, J. Shefner (2022)
MiToS and King’s staging as clinical outcome measures in ALS: a retrospective analysis of the FORTITUDE-ALS trialAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 24
Abstract Objective: To determine the target population and optimize the study design of the phase 3 clinical trial evaluating reldesemtiv in participants with amyotrophic lateral sclerosis (ALS). Methods: We evaluated the phase 2 study of reldesemtiv, FORTITUDE-ALS, to inform eligibility criteria and design features that would increase trial efficiency and reduce participant burden of the phase 3 trial. Results: In FORTITUDE-ALS, the effect of reldesemtiv was particularly evident among participants in the intermediate- and fast-progressing tertiles for pre-study disease progression. These participants most often had symptom onset ≤24 months and an ALS Functional Rating Scale-Revised (ALSFRS-R) total score ≤44 at baseline. Compared with the overall FORTITUDE-ALS population, the subgroup meeting these criteria declined by fewer ALSFRS-R points at 12 weeks (difference of least-squares mean [SE] versus placebo 1.84 [0.49] and 0.87 [0.35] for the overall population). These inclusion criteria will be used for the phase 3 clinical trial, COURAGE-ALS, in which the primary outcome is the change in ALSFRS-R total score at week 24. We also measure durable medical equipment use and evaluate strength in muscles expected to change rapidly. To reduce participant burden, study visits are often remote, and strength evaluation is simplified to reduce time and effort. Conclusions: In COURAGE-ALS, the phase 3 clinical trial to evaluate reldesemtiv, the sensitivity of detecting a potential treatment effect may be increased by defining eligibility criteria that limit the proportion of participants who have slower disease progression. Implementing remote visits and simplifying strength measurements will reduce both site and participant burden. ClinicalTrials.gov identifiers: NCT03160898 (FORTITUDE-ALS) and NCT04944784 (COURAGE-ALS)
Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration – Taylor & Francis
Published: Jul 3, 2023
Keywords: Amyotrophic lateral sclerosis; randomized clinical trial; reldesemtiv; ALSFRS-R
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