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Original Research

Comparing methods to combine functional loss and mortality in clinical trials for amyotrophic lateral sclerosis

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Pages 333-341 | Published online: 19 Mar 2018

Keep up to date with the latest research on this topic with citation updates for this article.

Read on this site (5)

Susana Pinto & Mamede De carvalho. (2021) Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 22:sup1, pages 9-13.
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Ruben P.A. van Eijk, Adriaan D. de Jongh, Stavros Nikolakopoulos, Christopher J. McDermott, Marinus J.C. Eijkemans, Kit C.B. Roes & Leonard H. van den Berg. (2021) An old friend who has overstayed their welcome: the ALSFRS-R total score as primary endpoint for ALS clinical trials. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 22:3-4, pages 300-307.
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Michael A. Van Es, Ruben P.A. Van Eijk, Tommy M. Bunte & Leonard H. Van Den Berg. (2020) A placebo-controlled trial to investigate the safety and efficacy of Penicillin G/Hydrocortisone in patients with ALS (PHALS trial). Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 21:7-8, pages 584-592.
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Ruben P.A. van Eijk, Tessa Kliest, Christopher J. McDermott, Kit C.B. Roes, Philip Van Damme, Adriano Chio, Markus Weber, Caroline Ingre, Philippe Corcia, Mònica Povedano, Evy Reviers, Michael A. van Es, Ammar Al-Chalabi, Orla Hardiman & Leonard H. van den Berg. (2020) TRICALS: creating a highway toward a cure. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 21:7-8, pages 496-501.
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Stephen A. Goutman, Morton B. Brown, Merit Cudkowicz, Nazem Atassi & Eva L. Feldman. (2019) ALS/SURV: a modification of the CAFS statistic. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 20:7-8, pages 576-583.
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Articles from other publishers (17)

Sean W. Willemse, Kit C. B. Roes, Philip Van Damme, Orla Hardiman, Caroline Ingre, Monica Povedano, Naomi R. Wray, Marleen Gijzen, Mirjam S. de Pagter, Koen C. Demaegd, Annemarie F. C. Janse, Roel G. Vink, Boudewijn T. H. M. Sleutjes, Adriano Chiò, Philippe Corcia, Evy Reviers, Ammar Al-Chalabi, Matthew C. Kiernan, Leonard H. van den Berg, Michael A. van Es & Ruben P. A. van Eijk. (2022) Lithium carbonate in amyotrophic lateral sclerosis patients homozygous for the C-allele at SNP rs12608932 in UNC13A: protocol for a confirmatory, randomized, group-sequential, event-driven, double-blind, placebo-controlled trial. Trials 23:1.
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Rob C. Van Wijk & Ulrika S. H. Simonsson. (2022) Finding the right hazard function for time‐to‐event modeling: A tutorial and Shiny application. CPT: Pharmacometrics & Systems Pharmacology 11:8, pages 991-1001.
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Ruben P.A. van Eijk, Kit C.B. Roes, Leonard H. van den Berg & Ying Lu. (2022) Joint modeling of endpoints can be used to answer various research questions in randomized clinical trials. Journal of Clinical Epidemiology 147, pages 32-39.
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Merit E. Cudkowicz & Jeremy M. Shefner. (2022) Regulatory Approval in ALS; When Is a Single Study Enough?. Annals of Neurology 91:6, pages 737-739.
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Paolo Tornese, Stefania Lalli, Antoniangela Cocco & Alberto Albanese. (2022) Review of disease-modifying drug trials in amyotrophic lateral sclerosis. Journal of Neurology, Neurosurgery & Psychiatry 93:5, pages 521-529.
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Ruben P.A. van Eijk, Kit C.B. Roes, Inez de Greef‐van der Sandt, Leonard H. van den Berg & Ying Lu. (2022) Functional Loss and Mortality in Randomized Clinical Trials for Amyotrophic Lateral Sclerosis: To Combine, or Not to Combine—That is the Estimand. Clinical Pharmacology & Therapeutics 111:4, pages 817-825.
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Sabrina Paganoni, James D. Berry, Melanie Quintana, Eric Macklin, Benjamin R. Saville, Michelle A. Detry, Marianne Chase, Alexander V. Sherman, Hong Yu, Kristin Drake, Jinsy Andrews, Jeremy Shefner, Lori B. Chibnik, Matteo Vestrucci, Merit E. Cudkowicz, Eric Macklin, Melanie Quintana, Ben Saville, Michelle A. Detry, Lori Chibnik, Marie Abele Bind, James Chan, Matteo Vestrucci, Eduardo Locatelli, Gustavo Alameda, Sabrina Paganoni, Merit Cudkowicz, Alexandra McCaffrey, James Berry, Suma Babu, Katie Nicholson, Jennifer Scalia, Zachary Simmons, James Grogan, Xiaowei Su, Mansoureh Mamarabadi, Daragh Heitzman, Mohamad Asaad Nasri, Jonathan Katz, Liberty Jenkins, Kevin Felice, Charles Whitaker, Senda Ajroud‐Driss, Adam Quick, Stephen Kolb, N. P. Sarah Heintzman, Stanley H. Appel, Ericka (Simpson) Greene, Jason Thonhoff, Sheetal Shroff, Bing Liao, Stephen A. Goutman, Eva L. Feldman, Timothy Miller, N. P. Amber Malcolm, Daniel S. Newman, Ximena Arcila‐Londono, Kara Steijlen, Margaret Ayo Owegi, Robert H. BrownJrJr, Mehdi Ghasemi, Joseph Americo M. Fernandes, Ezequiel Piccione, Pariwat Thaisetthawatkul, Michael D. Weiss, Nassim Rad, Hristelina Ilieva, Piera Pasinelli, N. P. Judith Guarnieri, Shafeeq Ladha, Bill Jacobsen, Robert Bowser, Jeremy Shefner, Laura Foster, Carlayne Jackson, Jeffrey Rosenfeld, Karthikeyan Bhuvaneswaran, Lauren Elman, Colin Quinn, Jinsy Andrews, N. P. Julia Yasek, Dominic Fee, Terry Heiman‐Patterson, Anahita Deboo, Andrea Swenson, Christopher Nance, Tuan Vu, Niraja Suresh, Jerrica Farias, Jim Caress, Michael Cartwright, Kourosh Rezania, Matthew Elliott, Seward Rutkove, Courtney McIlduff, Jeffrey A. Cohen, Elijah Stommel, Jonathan Glass, Christina Fournier, Eufrosina Young, James P. Wymer, Volkan Granit, Michael Benatar, Richard Lewis, Matthew Burford, Frank Diaz, Nicholas J. Maragakis, Lora Clawson, Alpa Uchil, Kristen Riley, Betsy Mosmiller, Paul Twydell, Namita A. Goyal, Omar Jawdat, Duaa Jabari, Jeffrey Statland, Edward J. Kasarskis, James W. Russell, Peter Jin, David Walk, Sam Maiser, Jaimin Shah, Bjorn Oskarsson, Richard Bedlack, Xiaoyan Li, I‐Hweii Amy Chen, Nicholas Olney, Tracy Bazan, Ghazala Hayat, Gary L. Pattee, Peter D. Donofrio, Amanda C. Peltier, Jennifer Martinez‐Thompson, Nathan, Kristin Johnson, Raghav Govindarajan, Vovanti Jones, Tulio Bertorini, Clotilde Lagier Tourenne, Ghazaleh Sadri Vakili, Marianne Chase, Alex Sherman, Hong Yu, Derek Dagostino, Scott Bocchio, Matthew Eydinov, Kenneth Faulconer, Olga Kharakozova, Alexander Korin, Ilya Novak, Ervin Sinani, Natalia Tarasenko, Prasha Vigneswaran, Igor Katsovskiy, Isaac Whitworth, Yusra Wahab, Catherine Gladden, Aileen Shaughnessy, Eric Tustison, Katie Jentoft, Joe Ostrow, Sangwon Lim, Genevive Changkuon, Annette De Mattos, Janae Patterson, Robin Scott, Precious Figueroa‐Szostek, Lindsay Pothier, Brittney Harkey, Sofia DiStefano, Erin Kennedy, Abbey Bailey, Nicole DiOrio, Jacob Feulner, Matt Davis, Jassira Gomes, Marisa DiMarzio, Jennifer Henrique, Molly Benson, Stacey Grabert, Tessa Garozzo, Meghan Hall, Rebecca Randall, Mariah Connolly, Diana De Santiago, Courtney Lavery, Gale Kittle, Jenny Hamilton, Patrick Bolger, Ahmed Fetouh, Cornelia Kamp, Julie Kennedy, Catherine Small, Allison Bulat, Kristin Drake, Andrew McGarry & Margherita Torti. (2022) Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development. Annals of Neurology 91:2, pages 165-175.
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Ruben P.A. van Eijk, Stavros Nikolakopoulos, Kit C.B. Roes, Lindsay Kendall, Steve S. Han, Arseniy Lavrov, Noam Epstein, Tessa Kliest, Adriaan D. de Jongh, Henk-Jan Westeneng, Ammar Al-Chalabi, Philip Van Damme, Orla Hardiman, Pamela J. Shaw, Christopher J. McDermott, Marinus J.C. Eijkemans & Leonard H. van den Berg. (2021) Innovating Clinical Trials for Amyotrophic Lateral Sclerosis. Neurology 97:11, pages 528-536.
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Siw Johannesen, J. Russell Huie, Bettina Budeus, Sebastian Peters, Anna M. Wirth, Sabine Iberl, Tina Kammermaier, Ines Kobor, Eva Wirkert, Sabrina Küspert, Marlene Tahedl, Jochen Grassinger, Tobias Pukrop, Armin Schneider, Ludwig Aigner, Wilhelm Schulte-Mattler, Gerhard Schuierer, Winfried Koch, Tim-Henrik Bruun, Adam R. Ferguson & Ulrich Bogdahn. (2021) Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis. Frontiers in Neurology 12.
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P. M. Brown, Tormod Rogne & Erik Solligård. (2020) The promise and pitfalls of composite endpoints in sepsis and COVID ‐19 clinical trials . Pharmaceutical Statistics 20:2, pages 413-417.
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Federico Ranieri, Sara Mariotto, Raffaele Dubbioso & Vincenzo Di Lazzaro. (2021) Brain Stimulation as a Therapeutic Tool in Amyotrophic Lateral Sclerosis: Current Status and Interaction With Mechanisms of Altered Cortical Excitability. Frontiers in Neurology 11.
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Namita A. Goyal, James D. Berry, Anthony Windebank, Nathan P. Staff, Nicholas J. Maragakis, Leonard H. Berg, Angela Genge, Robert Miller, Robert H. Baloh, Ralph Kern, Yael Gothelf, Chaim Lebovits & Merit Cudkowicz. (2020) Addressing heterogeneity in amyotrophic lateral sclerosis CLINICAL TRIALS. Muscle & Nerve 62:2, pages 156-166.
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Adriaan D de Jongh, Ruben P A van Eijk & Leonard H van den Berg. (2019) Evidence for a multimodal effect of riluzole in patients with ALS?. Journal of Neurology, Neurosurgery & Psychiatry 90:10, pages 1183-1184.
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Christopher J. McDermott. (2019) Clinical trials in amyotrophic lateral sclerosis. Current Opinion in Neurology 32:5, pages 758-763.
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Ruben P A van Eijk, James Rooney, Orla Hardiman & Leonard H van den Berg. (2019) Two heads are better than one: benefits of joint models for ALS trials. Journal of Neurology, Neurosurgery & Psychiatry 90:9, pages 1071-1072.
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Ruben P.A. van Eijk, Henk-Jan Westeneng, Stavros Nikolakopoulos, Iris E. Verhagen, Michael A. van Es, Marinus J.C. Eijkemans & Leonard H. van den Berg. (2019) Refining eligibility criteria for amyotrophic lateral sclerosis clinical trials. Neurology 92:5, pages e451-e460.
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James Rooney, Deirdre Murray, Anna Campion, Hannah Moloney, Rachel Tattersall, Mark Doherty, Michaela Hammond, Mark Heverin, Russell McLaughlin & Orla Hardiman. (2019) The C9orf72 expansion is associated with accelerated respiratory function decline in a large Amyotrophic Lateral Sclerosis cohort. HRB Open Research 2, pages 23.
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