ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma
Journal of Clinical Oncology2024Vol. 43(6), pp. 716–729
Citations Over TimeTop 1% of 2024 papers
Christopher L. Moertel, Angela C. Hirbe, Hans Shuhaiber, Kevin Bielamowicz, Alpa Sidhu, David Viskochil, Michael Weber, Armend Lokku, L. Mary Smith, Nicholas K. Foreman, Fouad Hajjar, Rene McNall‐Knapp, Lauren Weintraub, Reuben Antony, Andrea Franson, Julia Meade, David Schiff, Tobias Walbert, Prakash Ambady, Daniela A. Bota, Cynthia Campen, Gurcharanjeet Kaur, Laura J. Klesse, Stefania Maraka, Paul L. Moots, Kathryn Nevel, Miriam Bornhorst, Ana Aguilar-Bonilla, Sarah Chagnon, Nagma Dalvi, Punita Gupta, Ziad Khatib, Laura K. Metrock, Phioanh L. Nghiemphu, Ryan D. Roberts, Nathan Robison, Zsila Sadighi, Stacie Stapleton, Dusica Babovic‐Vuksanovic, Timothy R. Gershon, for the ReNeu Trial Investigators, Ahmed M. Raslan, Alpa Sidhu, Ana Aguilar-Bonilla, Andrea T. Franson, Andrew Walter, Angela C. Hirbe, Brian A. Van Tine, Carl Koschmann, Christopher L. Moertel, Cynthia Campen, Daniela A. Bota, David Schiff, David Viskochil, Dusica Babovic‐Vuksanovic, Fouad M. Hajjar, Gurcharanjeet Kaur, Hans Shuhaiber, Jamie K. Capal, John M. Slopis, Jonathan Gill, Julia Meade, Kathryn Nevel, Laura K. Metrock, Kevin Bielamowicz, Laura J. Klesse, Lauren Weintraub, Leia Nghiemphu, Lindsay Kilburn, Maciej M. Mrugała, Mary Lou Schmidt, Miriam Bornhorst, Nagma Dalvi, Nathan Robison, Nicholas K. Foreman, Paul L. Moots, Prakash Ambady, Punita Gupta, Radhika Dhamija, Rene McNall‐Knapp, Raju Antony, Ryan D. Roberts, Ryan Merrell, Sarah Chagnon, Stacie Stapleton, Stefania Maraka, Timothy R. Gershon, Tobias Walbert, Ziad Khatib, Zsila Sadighi
Abstract
In ReNeu, the largest multicenter NF1-PN trial reported to date, mirdametinib treatment demonstrated significant confirmed ORRs by BICR, deep and durable PN volume reductions, and early, sustained, and clinically meaningful improvement in pain and HRQOL. Mirdametinib was well-tolerated in adults and children.
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