Research Article
Cancer phase I clinical trials: efficient dose escalation with overdose control
First published: 04 December 1998
Citations: 323
Abstract
We describe an adaptive dose escalation scheme for use in cancer phase I clinical trials. The method is fully adaptive, makes use of all the information available at the time of each dose assignment, and directly addresses the ethical need to control the probability of overdosing. It is designed to approach the maximum tolerated dose as fast as possible subject to the constraint that the predicted proportion of patients who receive an overdose does not exceed a specified value. We conducted simulations to compare the proposed method with four up‐and‐down designs, two stochastic approximation methods, and with a variant of the continual reassessment method. The results showed the proposed method effective as a means to control the frequency of overdosing. Relative to the continual reassessment method, our scheme overdosed a smaller proportion of patients, exhibited fewer toxicities and estimated the maximum tolerated dose with comparable accuracy. When compared to the non‐parametric schemes, our method treated fewer patients at either subtherapeutic or severely toxic dose levels, treated more patients at optimal dose levels and estimated the maximum tolerated dose with smaller average bias and mean squared error. Hence, the proposed method is promising alternative to currently used cancer phase I clinical trial designs. © 1998 John Wiley & Sons, Ltd.
Citing Literature
Number of times cited according to CrossRef: 323
- Daniel S-W Tan, Natasha B Leighl, Gregory J Riely, James C-H Yang, Lecia V Sequist, Juergen Wolf, Takashi Seto, Enriqueta Felip, Santiago P Aix, Maud Jonnaert, Chun Pan, Eugene Y Tan, Jinnie Ko, Susan E Moody, Dong-Wan Kim, Safety and efficacy of nazartinib (EGF816) in adults with EGFR-mutant non-small-cell lung carcinoma: a multicentre, open-label, phase 1 study, The Lancet Respiratory Medicine, 10.1016/S2213-2600(19)30267-X, (2020).
- Shuang Li, Xian-Jin Xie, Daniel F. Heitjan, Flexible, rule-based dose escalation: The cohort-sequence design, Contemporary Clinical Trials Communications, 10.1016/j.conctc.2020.100541, (100541), (2020).
- José L. Jiménez, Sungjin Kim, Mourad Tighiouart, A Bayesian seamless phase I–II trial design with two stages for cancer clinical trials with drug combinations, Biometrical Journal, 10.1002/bimj.201900095, 62, 5, (1300-1314), (2020).
- Travis Culpepper, Joseph Young, Benjamin Wherley, Comparison of four warm‐season turfgrass species to natural rainfall or supplemental irrigation in a semiarid climate, Agrosystems, Geosciences & Environment, 10.1002/agg2.20011, 3, 1, (2020).
- John G. Nutt, Carolin Curtze, Amie Hiller, Shannon Anderson, Paul S. Larson, Amber D. Van Laar, R. Mark Richardson, Marin E. Thompson, Alexander Sedkov, Mika Leinonen, Bernard Ravina, Krystof S. Bankiewicz, Chadwick W. Christine, Aromatic L‐Amino Acid Decarboxylase Gene Therapy Enhances Levodopa Response in Parkinson's Disease, Movement Disorders, 10.1002/mds.27993, 35, 5, (851-858), (2020).
- Abdelaziz Nilahyane, Rajan Ghimire, Vesh R. Thapa, Upendra M. Sainju, Cover crop effects on soil carbon dioxide emissions in a semiarid cropping system, Agrosystems, Geosciences & Environment, 10.1002/agg2.20012, 3, 1, (2020).
- Shelemyahu Zacks, Shelemyahu Zacks, Sequential Search of an Optimal Dosage, The Career of a Research Statistician, 10.1007/978-3-030-39434-9_10, (149-158), (2020).
- D. Dinart, J. Fraisse, D. Tosi, A. Mauguen, C. Touraine, S. Gourgou, M. C. Le Deley, C. Bellera, C. Mollevi, GUIP1: a R package for dose escalation strategies in phase I cancer clinical trials, BMC Medical Informatics and Decision Making, 10.1186/s12911-020-01149-3, 20, 1, (2020).
- Mourad Tighiouart, Dose Finding for Drug Combinations, Principles and Practice of Clinical Trials, 10.1007/978-3-319-52677-5, (1-29), (2020).
- Guosheng Yin, Zhao Yang, Fractional design: An alternative paradigm for late-onset toxicities in oncology dose-finding studies, Contemporary Clinical Trials Communications, 10.1016/j.conctc.2020.100650, (100650), (2020).
- Chia-Chi Lin, Novel Trial Designs for Early Phase Clinical Trials, Phase I Oncology Drug Development, 10.1007/978-3-030-47682-3, (85-93), (2020).
- Nancy Flournoy, José Moler, Fernando Plo, Performance Measures in Dose‐Finding Experiments, International Statistical Review, 10.1111/insr.12363, 0, 0, (2020).
- Roberto Fumagalli, Buyer Beware: A Critique of Leading Virtue Ethics Defenses of Markets, Journal of Social Philosophy, 10.1111/josp.12336, 0, 0, (2020).
- Jean-Pierre Delord, Guillem Argilés, Jerôme Fayette, Lori Wirth, Stefan Kasper, Salvatore Siena, Ricard Mesia, Rossana Berardi, Andrés Cervantes, Jeroen Dekervel, Sylvia Zhao, Yongjian Sun, Huai-Xiang Hao, Ralph Tiedt, Sergio Vicente, Andrea Myers, Lillian L. Siu, A phase 1b study of the MET inhibitor capmatinib combined with cetuximab in patients with MET-positive colorectal cancer who had progressed following anti-EGFR monoclonal antibody treatment, Investigational New Drugs, 10.1007/s10637-020-00928-z, (2020).
- Márcio A. Diniz, Sungjin Kim, Mourad Tighiouart, A Bayesian Adaptive Design in Cancer Phase I Trials Using Dose Combinations with Ordinal Toxicity Grades, Stats, 10.3390/stats3030017, 3, 3, (221-238), (2020).
- Yanhong Zhou, Ruobing Li, Fangrong Yan, J. Jack Lee, Ying Yuan, A comparative study of Bayesian optimal interval (BOIN) design with interval 3 + 3 (i3 + 3) design for phase I oncology dose-finding trials, Statistics in Biopharmaceutical Research, 10.1080/19466315.2020.1811147, (1-21), (2020).
- Jun Yin, Ying Yuan, Checkerboard: a Bayesian efficacy and toxicity interval design for phase I/II dose-finding trials, Journal of Biopharmaceutical Statistics, 10.1080/10543406.2020.1815033, (1-20), (2020).
- Oleksandr Sverdlov, Yevgen Ryeznik, Weng Kee Wong, On Optimal Designs for Clinical Trials: An Updated Review, Journal of Statistical Theory and Practice, 10.1007/s42519-019-0073-4, 14, 1, (2019).
- Muna Qayed, Thomas Cash, Mourad Tighiouart, Tobey J. MacDonald, Kelly C. Goldsmith, Rachel Tanos, Leslie Kean, Benjamin Watkins, Yvonne Suessmuth, Cynthia Wetmore, Howard M. Katzenstein, A phase I study of sirolimus in combination with metronomic therapy (CHOAnome) in children with recurrent or refractory solid and brain tumors, Pediatric Blood & Cancer, 10.1002/pbc.28134, 67, 4, (2019).
- Aditya Bardia, Mrinal Gounder, Jordi Rodon, Filip Janku, Martijn P. Lolkema, Joe J. Stephenson, Philippe L. Bedard, Martin Schuler, Cristiana Sessa, Patricia LoRusso, Michael Thomas, Heiko Maacke, Helen Evans, Yongjian Sun, Daniel S.W. Tan, Phase Ib Study of Combination Therapy with MEK Inhibitor Binimetinib and Phosphatidylinositol 3‐Kinase Inhibitor Buparlisib in Patients with Advanced Solid Tumors with RAS/RAF Alterations, The Oncologist, 10.1634/theoncologist.2019-0297, 25, 1, (e160-e169), (2019).
- Ruitao Lin, Peter F. Thall, Ying Yuan, An adaptive trial design to optimize dose‐schedule regimes with delayed outcomes, Biometrics, 10.1111/biom.13116, 76, 1, (304-315), (2019).
- Pavel Mozgunov, Thomas Jaki, Improving safety of the continual reassessment method via a modified allocation rule, Statistics in Medicine, 10.1002/sim.8450, 39, 7, (906-922), (2019).
- Yung‐Jue Bang, Wu‐Chou Su, Martin Schuler, Do‐Hyun Nam, Wan Teck Lim, Todd M. Bauer, Analia Azaro, Ronnie Tung Ping Poon, David Hong, Chia‐Chi Lin, Mikhail Akimov, Samson Ghebremariam, Sylvia Zhao, Monica Giovannini, Brigette Ma, Phase 1 study of capmatinib in MET‐positive solid tumor patients: Dose escalation and expansion of selected cohorts, Cancer Science, 10.1111/cas.14254, 111, 2, (536-547), (2019).
- Takashi Daimon, Akihiro Hirakawa, Shigeyuki Matsui, Takashi Daimon, Akihiro Hirakawa, Shigeyuki Matsui, Model-Based Designs Considering Toxicity Alone, Dose-Finding Designs for Early-Phase Cancer Clinical Trials, 10.1007/978-4-431-55585-8_3, (33-79), (2019).
- Daniel J. DeAngelo, Alison R. Walker, Richard F. Schlenk, Jorge Sierra, Bruno C. Medeiros, Enrique M. Ocio, Christoph Röllig, Stephen A. Strickland, Felicitas Thol, Sue-zette Valera, Kohinoor Dasgupta, Noah Berkowitz, Robert K. Stuart, Safety and efficacy of oral panobinostat plus chemotherapy in patients aged 65 years or younger with high-risk acute myeloid leukemia, Leukemia Research, 10.1016/j.leukres.2019.106197, (106197), (2019).
- Li Liu, Lei Gao, Glen Laird, Bayesian Dose Escalation Study Design with Consideration of Both Early and Late Onset Toxicity, Contemporary Biostatistics with Biopharmaceutical Applications, 10.1007/978-3-030-15310-6_12, (225-238), (2019).
- Bernard North, Hemant Mahendrakumar Kocher, Peter Sasieni, A new pragmatic design for dose escalation in phase 1 clinical trials using an adaptive continual reassessment method, BMC Cancer, 10.1186/s12885-019-5801-3, 19, 1, (2019).
- I. Domenicano, S. Ventz, M. Cellamare, R. H. Mak, L. Trippa, Bayesian uncertainty‐directed dose finding designs, Journal of the Royal Statistical Society: Series C (Applied Statistics), 10.1111/rssc.12355, 68, 5, (1393-1410), (2019).
- Andrew G. Chapple, Peter F. Thall, A hybrid phase I‐II/III clinical trial design allowing dose re‐optimization in phase III, Biometrics, 10.1111/biom.12994, 75, 2, (371-381), (2019).
- Taito Esaki, Fumihiko Hirai, Akitaka Makiyama, Takashi Seto, Hideaki Bando, Yoichi Naito, Kiyotaka Yoh, Kae Ishihara, Tomoyuki Kakizume, Kazuto Natsume, Andrea Myers, Toshihiko Doi, Phase I dose‐escalation study of capmatinib (INC280) in Japanese patients with advanced solid tumors, Cancer Science, 10.1111/cas.13956, 110, 4, (1340-1351), (2019).
- Timothy P. Hughes, Michael J. Mauro, Jorge E. Cortes, Hironobu Minami, Delphine Rea, Daniel J. DeAngelo, Massimo Breccia, Yeow-Tee Goh, Moshe Talpaz, Andreas Hochhaus, Philipp le Coutre, Oliver Ottmann, Michael C. Heinrich, Juan L. Steegmann, Michael W.N. Deininger, Jeroen J.W.M. Janssen, Francois-Xavier Mahon, Yosuke Minami, David Yeung, David M. Ross, Martin S. Tallman, Jae H. Park, Brian J. Druker, David Hynds, Yuyan Duan, Christophe Meille, Florence Hourcade-Potelleret, K. Gary Vanasse, Fabian Lang, Dong-Wook Kim, Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure, New England Journal of Medicine, 10.1056/NEJMoa1902328, 381, 24, (2315-2326), (2019).
- Enriqueta Felip, Filippo G. de Braud, Michela Maur, Herbert H. Loong, Alice Tsang Shaw, Johan F. Vansteenkiste, Thomas John, Geoffrey Liu, Martijn P. Lolkema, Giovanni Selvaggi, Vanessa Giannone, Pilar Cazorla, Jason Baum, O. Alejandro Balbin, Luojun (Victor) Wang, Yvonne Y. Lau, Jeffrey W. Scott, Daniel Shao-Weng Tan, Ceritinib plus Nivolumab in Patients with Advanced ALK-Rearranged Non–Small Cell Lung Cancer: Results of an Open-Label, Multicenter, Phase 1B Study, Journal of Thoracic Oncology, 10.1016/j.jtho.2019.10.006, (2019).
- Chi Kin Lam, Ruitao Lin, Guosheng Yin, Non‐parametric overdose control for dose finding in drug combination trials, Journal of the Royal Statistical Society: Series C (Applied Statistics), 10.1111/rssc.12349, 68, 4, (1111-1130), (2019).
- Ajlan Atasoy, Murielle Mauer, Improving Clinical Trial Design in Gastrointestinal Oncology, Textbook of Gastrointestinal Oncology, 10.1007/978-3-030-18890-0, (493-507), (2019).
- Lingyun Ji, Juan Pablo Lewinger, Mark Krailo, Susan Groshen, David V. Conti, Shahab Asgharzadeh, Richard Sposto, Improvements to the Escalation with Overdose Control design and a comparison with the restricted Continual Reassessment Method, Pharmaceutical Statistics, 10.1002/pst.1955, 18, 6, (659-670), (2019).
- Ying Yuan, J. Jack Lee, Susan G. Hilsenbeck, Model-Assisted Designs for Early-Phase Clinical Trials: Simplicity Meets Superiority, JCO Precision Oncology, 10.1200/PO.19.00032, 3, (1-12), (2019).
- Márcio Augusto Diniz, Mourad Tighiouart, André Rogatko, Comparison between continuous and discrete doses for model based designs in cancer dose finding, PLOS ONE, 10.1371/journal.pone.0210139, 14, 1, (e0210139), (2019).
- Weijia Zhang, Xikui Wang, Po Yang, A new design of the continual reassessment method, Communications in Statistics - Simulation and Computation, 10.1080/03610918.2019.1592191, (1-10), (2019).
- Haiyan Zheng, Lisa V Hampson, Simon Wandel, A robust Bayesian meta-analytic approach to incorporate animal data into phase I oncology trials, Statistical Methods in Medical Research, 10.1177/0962280218820040, (096228021882004), (2019).
- Marc S. Raab, Sheeba K. Thomas, Enrique M. Ocio, Andreas Guenther, Yeow-Tee Goh, Moshe Talpaz, Nicolas Hohmann, Sylvia Zhao, Fang Xiang, Carl Simon, K. Gary Vanasse, Shaji K. Kumar, The first-in-human study of the pan-PIM kinase inhibitor PIM447 in patients with relapsed and/or refractory multiple myeloma, Leukemia, 10.1038/s41375-019-0482-0, (2019).
- Rebecca S Slack Tidwell, S Andrew Peng, Minxing Chen, Diane D Liu, Ying Yuan, J Jack Lee, Bayesian clinical trials at The University of Texas MD Anderson Cancer Center: An update, Clinical Trials, 10.1177/1740774519871471, (174077451987147), (2019).
- Puntipa Wanitjirattikal, Chenyang Shi, A Bayesian zero-inflated binomial regression and its application in dose-finding study, Journal of Biopharmaceutical Statistics, 10.1080/10543406.2019.1684313, (1-12), (2019).
- Nolan A. Wages, Evan Bagley, Evaluation of irrational dose assignment definitions using the continual reassessment method, Clinical Trials, 10.1177/1740774519873316, (174077451987331), (2019).
- Boris Böll, Annette Plütschow, Carolin Bürkle, Johannes Atta, Michael Pfreundschuh, Michaela Feuring‐Buske, Martin Vogelhuber, Martin Sökler, Dennis A. Eichenauer, Indra Thielen, Bastian Tresckow, Michael Fuchs, Andreas Engert, Peter Borchmann, Doxorubicin, vinblastine, dacarbazine and lenalidomide for older Hodgkin lymphoma patients: final results of a German Hodgkin Study Group (GHSG) phase‐I trial, British Journal of Haematology, 10.1111/bjh.15741, 185, 1, (42-52), (2018).
- Rongji Mu, Ying Yuan, Jin Xu, Sumithra J. Mandrekar, Jun Yin, gBOIN: a unified model‐assisted phase I trial design accounting for toxicity grades, and binary or continuous end points, Journal of the Royal Statistical Society: Series C (Applied Statistics), 10.1111/rssc.12263, 68, 2, (289-308), (2018).
- Mourad Tighiouart, Two‐stage design for phase I–II cancer clinical trials using continuous dose combinations of cytotoxic agents, Journal of the Royal Statistical Society: Series C (Applied Statistics), 10.1111/rssc.12294, 68, 1, (235-250), (2018).
- Jie Shen, Brandon Swift, Richard Mamelok, Samuel Pine, John Sinclair, Mayssa Attar, Design and Conduct Considerations for First‐in‐Human Trials, Clinical and Translational Science, 10.1111/cts.12582, 12, 1, (6-19), (2018).
- Jose L. Jimenez, Mourad Tighiouart, Mauro Gasparini, Cancer phase I trial design using drug combinations when a fraction of dose limiting toxicities is attributable to one or more agents, Biometrical Journal, 10.1002/bimj.201700166, 61, 2, (319-332), (2018).
- Simon Wandel, Satrajit Roychoudhury, Statistical Considerations in Phase I Oncology Trials, Statistical Approaches in Oncology Clinical Development, 10.1201/9781315154435, (5-41), (2018).
- Zhengjia Chen, Youyun Zheng, Zhibo Wang, Michael Kutner, Walter J. Curran, Jeanne Kowalski, Interactive calculator for operating characteristics of phase I cancer clinical trials using standard 3+3 designs, Contemporary Clinical Trials Communications, 10.1016/j.conctc.2018.10.006, (2018).
- Akihiro Hirakawa, Hiroyuki Sato, Takashi Daimon, Shigeyuki Matsui, Akihiro Hirakawa, Hiroyuki Sato, Takashi Daimon, Shigeyuki Matsui, Dose Finding in Phase I Cancer Trials, Modern Dose-Finding Designs for Cancer Phase I Trials: Drug Combinations and Molecularly Targeted Agents, 10.1007/978-4-431-55573-5_1, (1-7), (2018).
- Márcio Augusto Diniz, Sungjin Kim, Mourad Tighiouart, A Bayesian Adaptive Design in Cancer Phase I Trials Using Dose Combinations in the Presence of a Baseline Covariate, Journal of Probability and Statistics, 10.1155/2018/8654173, 2018, (1-11), (2018).
- Jordi Rodon, Alejandro Pérez-Fidalgo, Ian E. Krop, Howard Burris, Angel Guerrero-Zotano, Carolyn D. Britten, Carlos Becerra, Jan Schellens, Donald A. Richards, Martin Schuler, Maysa Abu-Khalaf, Faye M. Johnson, Malcolm Ranson, Jeff Edenfield, Antonio P. Silva, Wolfgang Hackl, Cornelia Quadt, David Demanse, Vincent Duval, Jose Baselga, Phase 1/1b dose escalation and expansion study of BEZ235, a dual PI3K/mTOR inhibitor, in patients with advanced solid tumors including patients with advanced breast cancer, Cancer Chemotherapy and Pharmacology, 10.1007/s00280-018-3610-z, 82, 2, (285-298), (2018).
- Md. Moniruzzaman Moni, M. Iftakhar Alam, Patient-specific dose finding in phase I clinical trials, Journal of Applied Statistics, 10.1080/02664763.2018.1428735, 45, 14, (2607-2618), (2018).
- Elizabeth Garrett-Mayer, Nathaniel O’Connell, The Evolution of Phase I Trials, Past, Present, and Future, Novel Designs of Early Phase Trials for Cancer Therapeutics, 10.1016/B978-0-12-812512-0.00003-8, (17-32), (2018).
- Louise Carter, Ciara O’Brien, Emma Dean, Natalie Cook, How to Design Phase I Trials in Oncology, Methods and Biostatistics in Oncology, 10.1007/978-3-319-71324-3, (165-187), (2018).
- Khanh Do, Chris H. Takimoto, Shivaani Kummar, Changing Landscape of Early Phase Clinical Trials, Novel Designs of Early Phase Trials for Cancer Therapeutics, 10.1016/B978-0-12-812512-0.00001-4, (1-6), (2018).
- M Ochoa de Olza, M Oliva, C Hierro, I Matos, J Martin-Liberal, E Garralda, Early-drug development in the era of immuno-oncology: are we ready to face the challenges?, Annals of Oncology, 10.1093/annonc/mdy225, 29, 8, (1727-1740), (2018).
- Thomas M. Braun, Motivating sample sizes in adaptive Phase I trials via Bayesian posterior credible intervals, Biometrics, 10.1111/biom.12872, 74, 3, (1065-1071), (2018).
- Amy Cotterill, Thomas Jaki, Dose‐escalation strategies which use subgroup information, Pharmaceutical Statistics, 10.1002/pst.1860, 17, 5, (414-436), (2018).
- Ruitao Lin, Guosheng Yin, Uniformly most powerful Bayesian interval design for phase I dose‐finding trials, Pharmaceutical Statistics, 10.1002/pst.1889, 17, 6, (710-724), (2018).
- Andrew G. Chapple, Peter F. Thall, Subgroup‐specific dose finding in phase I clinical trials based on time to toxicity allowing adaptive subgroup combination, Pharmaceutical Statistics, 10.1002/pst.1891, 17, 6, (734-749), (2018).
- Heng Zhou, Thomas A. Murray, Haitao Pan, Ying Yuan, Comparative review of novel model‐assisted designs for phase I clinical trials, Statistics in Medicine, 10.1002/sim.7674, 37, 14, (2208-2222), (2018).
- Efehan Ulas, Filiz Karaman, Tuba Koc, Impact of different model structure and prior distribution in continual reassessment method, Communications in Statistics - Simulation and Computation, 10.1080/03610918.2018.1476698, (1-15), (2018).
- Yukio Kobayashi, Wataru Munakata, Michinori Ogura, Toshiki Uchida, Masafumi Taniwaki, Tsutomu Kobayashi, Fumika Shimada, Masataka Yonemura, Fumiko Matsuoka, Takeshi Tajima, Kimikazu Yakushijin, Hironobu Minami, Phase I study of panobinostat and 5-azacitidine in Japanese patients with myelodysplastic syndrome or chronic myelomonocytic leukemia, International Journal of Hematology, 10.1007/s12185-017-2327-9, 107, 1, (83-91), (2017).
- Mourad Tighiouart, Galen Cook-Wiens, André Rogatko, A Bayesian adaptive design for cancer phase I trials using a flexible range of doses, Journal of Biopharmaceutical Statistics, 10.1080/10543406.2017.1372774, 28, 3, (562-574), (2017).
- Toshihiko Doi, Becker Hewes, Tomoyuki Kakizume, Takeshi Tajima, Norifumi Ishikawa, Yasuhide Yamada, Phase I study of single‐agent ribociclib in Japanese patients with advanced solid tumors, Cancer Science, 10.1111/cas.13428, 109, 1, (193-198), (2017).
- Bo Huang, Some statistical considerations in the clinical development of cancer immunotherapies, Pharmaceutical Statistics, 10.1002/pst.1835, 17, 1, (49-60), (2017).
- Sharon B Love, Sarah Brown, Christopher J Weir, Chris Harbron, Christina Yap, Birgit Gaschler-Markefski, James Matcham, Louise Caffrey, Christopher McKevitt, Sally Clive, Charlie Craddock, James Spicer, Victoria Cornelius, Embracing model-based designs for dose-finding trials, British Journal of Cancer, 10.1038/bjc.2017.186, 117, 3, (332-339), (2017).
- Chunyan Cai, Mohammad H. Rahbar, Md Monir Hossain, Ying Yuan, Nicole R. Gonzales, A placebo-controlled Bayesian dose finding design based on continuous reassessment method with application to stroke research, Contemporary Clinical Trials Communications, 10.1016/j.conctc.2017.05.002, 7, (11-17), (2017).
- Ciara O’Brien, Louise Carter, Natalie Cook, Emma Dean, Novel Early Phase Clinical Trial Design in Oncology, Pharmaceutical Medicine, 10.1007/s40290-017-0205-7, 31, 5, (297-307), (2017).
- Kerry Lynn Reynolds, Philippe L. Bedard, Se-Hoon Lee, Chia-Chi Lin, Josep Tabernero, Maria Alsina, Ezra Cohen, José Baselga, George Blumenschein, Donna M. Graham, Ignacio Garrido-Laguna, Dejan Juric, Sunil Sharma, Ravi Salgia, Abdelkader Seroutou, Xianbin Tian, Rose Fernandez, Alex Morozov, Qing Sheng, Thiruvamoor Ramkumar, Angela Zubel, Yung-Jue Bang, A phase I open-label dose-escalation study of the anti-HER3 monoclonal antibody LJM716 in patients with advanced squamous cell carcinoma of the esophagus or head and neck and HER2-overexpressing breast or gastric cancer, BMC Cancer, 10.1186/s12885-017-3641-6, 17, 1, (2017).
- Mark W Kieran, Julia Chisholm, Michela Casanova, Alba A Brandes, Isabelle Aerts, Eric Bouffet, Simon Bailey, Sarah Leary, Tobey J MacDonald, Francoise Mechinaud, Kenneth J Cohen, Riccardo Riccardi, Warren Mason, Darren Hargrave, Stacey Kalambakas, Priya Deshpande, Feng Tai, Eunju Hurh, Birgit Geoerger, Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma, Neuro-Oncology, 10.1093/neuonc/nox109, 19, 11, (1542-1552), (2017).
- G Garcia-Manero, M A Sekeres, M Egyed, M Breccia, C Graux, J D Cavenagh, H Salman, A Illes, P Fenaux, D J DeAngelo, R Stauder, K Yee, N Zhu, J-H Lee, D Valcarcel, A MacWhannell, Z Borbenyi, L Gazi, S Acharyya, S Ide, M Marker, O G Ottmann, A phase 1b/2b multicenter study of oral panobinostat plus azacitidine in adults with MDS, CMML or AML with ⩽30% blasts, Leukemia, 10.1038/leu.2017.159, 31, 12, (2799-2806), (2017).
- Emily V. Dressler, Donglin Yan, John O’Quigley, Alexia Iasonos, Björn Bornkamp, Model-Based Designs for Safety, Handbook of Methods for Designing, Monitoring, and Analyzing Dose-Finding Trials, 10.1201/9781315151984, (27-44), (2017).
- Joseph S. Koopmeiners, Andrew Wey, The Randomized CRM: An Approach to Overcoming the Long-Memory Property of the CRM, Journal of Biopharmaceutical Statistics, 10.1080/10543406.2017.1293076, 27, 6, (1028-1042), (2017).
- Lorenzo Capretto, Gerard Byrne, Sarah Trenfield, Lee Dowden, Steven Booth, Formulation, Analytical, and Regulatory Strategies for First‐in‐Human Clinical Trials, Oral Formulation Roadmap from Early Drug Discovery to Development, 10.1002/9781118907894, (165-241), (2017).
- Moreno Ursino, Sarah Zohar, Frederike Lentz, Corinne Alberti, Tim Friede, Nigel Stallard, Emmanuelle Comets, Dose‐finding methods for Phase I clinical trials using pharmacokinetics in small populations, Biometrical Journal, 10.1002/bimj.201600084, 59, 4, (804-825), (2017).
- Sheila M. Bird, Rosemary A. Bailey, Andrew P. Grieve, Stephen Senn, Statistical issues in first‐in‐human studies on BIA 10‐2474: Neglected comparison of protocol against practice, Pharmaceutical Statistics, 10.1002/pst.1801, 16, 2, (100-106), (2017).
- Wai Yin Yeung, Bruno Reigner, Ulrich Beyer, Cheikh Diack, Daniel Sabanés bové, Giuseppe Palermo, Thomas Jaki, Bayesian adaptive dose‐escalation designs for simultaneously estimating the optimal and maximum safe dose based on safety and efficacy, Pharmaceutical Statistics, 10.1002/pst.1818, 16, 6, (396-413), (2017).
- Graham M. Wheeler, Michael J. Sweeting, Adrian P. Mander, Toxicity‐dependent feasibility bounds for the escalation with overdose control approach in phase I cancer trials, Statistics in Medicine, 10.1002/sim.7280, 36, 16, (2499-2513), (2017).
- Brenda Gaydos, Michael Krams, Inna Perevozskaya, Frank Bred, Qing Liu, Paul Gallo, Don Berry, Christy Chuang-Stein, José Pinheiro, Alun Bedding, Adaptive Dose-Response Studies, Drug Information Journal, 10.1177/216847900604000411, 40, 4, (451-461), (2017).
- Vladimir Dragalin, Adaptive Designs: Terminology and Classification, Drug Information Journal, 10.1177/216847900604000408, 40, 4, (425-435), (2017).
- Kristen M Cunanan, Joseph S Koopmeiners, Hierarchical models for sharing information across populations in phase I dose-escalation studies, Statistical Methods in Medical Research, 10.1177/0962280217703812, (096228021770381), (2017).
- Zhengjia Chen, Zheng Li, Run Zhuang, Ying Yuan, Michael Kutner, Taofeek Owonikoko, Walter J. Curran, Jeanne Kowalski, Adaptive Estimation of Personalized Maximum Tolerated Dose in Cancer Phase I Clinical Trials Based on All Toxicities and Individual Genomic Profile, PLOS ONE, 10.1371/journal.pone.0170187, 12, 1, (e0170187), (2017).
- Jocelyn Gal, Gérard Milano, Jean-Marc Ferrero, Esma Saâda-Bouzid, Julien Viotti, Sylvie Chabaud, Paul Gougis, Christophe Le Tourneau, Renaud Schiappa, Agnes Paquet, Emmanuel Chamorey, Optimizing drug development in oncology by clinical trial simulation: Why and how?, Briefings in Bioinformatics, 10.1093/bib/bbx055, (2017).
- Rongji Mu, Jin Xu, A new Bayesian dose finding design for drug combination trials, Statistics in Biopharmaceutical Research, 10.1080/19466315.2017.1388834, (0-0), (2017).
- Graham M. Wheeler, Incoherent dose-escalation in phase I trials using the escalation with overdose control approach, Statistical Papers, 10.1007/s00362-016-0790-7, 59, 2, (801-811), (2016).
- Shunji Takahashi, Takayuki Kobayashi, Junichi Tomomatsu, Yoshinori Ito, Hisanobu Oda, Tatsuhiro Kajitani, Tomoyuki Kakizume, Takeshi Tajima, Hiromi Takeuchi, Heiko Maacke, Taito Esaki, LJM716 in Japanese patients with head and neck squamous cell carcinoma or HER2-overexpressing breast or gastric cancer, Cancer Chemotherapy and Pharmacology, 10.1007/s00280-016-3214-4, 79, 1, (131-138), (2016).
- Alexina J. Mason, Juan Gonzalez‐Maffe, Killian Quinn, Nicki Doyle, Ken Legg, Peter Norsworthy, Roy Trevelion, Alan Winston, Deborah Ashby, Developing a Bayesian adaptive design for a phase I clinical trial: a case study for a novel HIV treatment, Statistics in Medicine, 10.1002/sim.7169, 36, 5, (754-771), (2016).
- Mourad Tighiouart, Quanlin Li, André Rogatko, A Bayesian adaptive design for estimating the maximum tolerated dose curve using drug combinations in cancer phase I clinical trials, Statistics in Medicine, 10.1002/sim.6961, 36, 2, (280-290), (2016).
- Nolan A. Wages, Identifying a maximum tolerated contour in two‐dimensional dose finding, Statistics in Medicine, 10.1002/sim.6918, 36, 2, (242-253), (2016).
- Haitao Pan, Ying Yuan, A default method to specify skeletons for Bayesian model averaging continual reassessment method for phase I clinical trials, Statistics in Medicine, 10.1002/sim.6941, 36, 2, (266-279), (2016).
- Andrew P. Grieve, Response‐adaptive clinical trials: case studies in the medical literature, Pharmaceutical Statistics, 10.1002/pst.1778, 16, 1, (64-86), (2016).
- Bo Huang, Paul Bycott, Enayet Talukder, Novel dose-finding designs and considerations on practical implementations in oncology clinical trials, Journal of Biopharmaceutical Statistics, 10.1080/10543406.2016.1148715, 27, 1, (44-55), (2016).
- Laeeq Malik, Steven Weitman, Overview of Oncology Drug Development, Pharmacokinetics in Drug Development, 10.1007/978-3-319-39053-6, (1-27), (2016).
- Adelaide Doussau, Birgit Geoerger, Irene Jiménez, Xavier Paoletti, Innovations for phase I dose-finding designs in pediatric oncology clinical trials, Contemporary Clinical Trials, 10.1016/j.cct.2016.01.009, 47, (217-227), (2016).
- M. Iftakhar Alam, A comparison between the continual reassessment method and D-optimum design for dose finding in phase I clinical trials, Biometrical Letters, 10.1515/bile-2016-0006, 53, 2, (69-82), (2016).
- Hironobu Minami, Yuichi Ando, Brigette Buig Yue Ma, Jih‐ Hsiang Lee, Hiroyuki Momota, Yutaka Fujiwara, Leung Li, Koichi Fukino, Koji Ito, Takeshi Tajima, Asuka Mori, Chia‐Chi Lin, Phase I, multicenter, open‐label, dose‐escalation study of sonidegib in Asian patients with advanced solid tumors, Cancer Science, 10.1111/cas.13022, 107, 10, (1477-1483), (2016).
- Anastasia Ivanova, Yunfei Wang, Matthew C. Foster, The rapid enrollment design for Phase I clinical trials, Statistics in Medicine, 10.1002/sim.6886, 35, 15, (2516-2524), (2016).
- See more
