Skip to main content Accessibility help
×
Hostname: page-component-848d4c4894-pjpqr Total loading time: 0 Render date: 2024-06-16T10:43:49.054Z Has data issue: false hasContentIssue false

Part IV - Case Studies of Adaptive Trial Designs and Master Protocols

Published online by Cambridge University Press:  20 March 2023

Jay J. H. Park
Affiliation:
McMaster University, Ontario
Edward J. Mills
Affiliation:
McMaster University, Ontario
J. Kyle Wathen
Affiliation:
Cytel, Cambridge, Massachusetts
Get access

Summary

Image of the first page of this content. For PDF version, please use the ‘Save PDF’ preceeding this image.'
Type
Chapter
Information
Publisher: Cambridge University Press
Print publication year: 2023

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

References

Zajicek, JP, Hobart, JC, Slade, A, et al. Multiple sclerosis and extract of cannabis: results of the MUSEC trial. J Neurol Neurosurg Psychiatry. 2012;83(11):1125–32.CrossRefGoogle ScholarPubMed
Bethoux, F, Marrie, RA. A cross-sectional study of the impact of spasticity on daily activities in multiple sclerosis. Patient. 2016;9(6):537–46.Google Scholar
O’Brien, PC, Fleming, TR. A multiple testing procedure for clinical trials. Biometrics. 1979;35(3):549–56.Google Scholar
Dimairo, M, Pallmann, P, Wason, J, et al. The Adaptive designs CONSORT Extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design. BMJ. 2020;369:m115.Google Scholar
Dimairo, M, Pallmann, P, Wason, J, et al. The adaptive designs CONSORT extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design. Trials. 2020;21(1):528.CrossRefGoogle ScholarPubMed
Sevransky, JE, Rothman, RE, Hager, DN, et al. Effect of vitamin C, thiamine, and hydrocortisone on ventilator- and vasopressor-free days in patients with sepsis: the VICTAS randomized clinical trial. JAMA. 2021;325(8):742–50.CrossRefGoogle ScholarPubMed
Lindsell, CJ, McGlothlin, A, Nwosu, S, et al. Update to the Vitamin C, Thiamine and Steroids in Sepsis (VICTAS) protocol: statistical analysis plan for a prospective, multicenter, double-blind, adaptive sample size, randomized, placebo-controlled, clinical trial. Trials. 2019;20(1):670.Google Scholar
Hager, DN, Hooper, MH, Bernard, GR, et al. The Vitamin C, Thiamine and Steroids in Sepsis (VICTAS) Protocol: a prospective, multi-center, double-blind, adaptive sample size, randomized, placebo-controlled, clinical trial. Trials. 2019;20(1):197.Google Scholar
Bauchner, H, Fontanarosa, PB, Golub, RM. Funding and DSMB membership in the VICTAS clinical trial. JAMA. 2021;325(8):751–2.Google Scholar
Galloway, DA, Laimins, LA. Human papillomaviruses: shared and distinct pathways for pathogenesis. Curr Opin Virol. 2015;14:8792.CrossRefGoogle ScholarPubMed
Plummer, M, de Martel, C, Vignat, J, et al. Global burden of cancers attributable to infections in 2012: a synthetic analysis. Lancet Glob Health. 2016;4(9):e609–16.CrossRefGoogle ScholarPubMed
Joura, EA, Leodolter, S, Hernandez-Avila, M, et al. Efficacy of a quadrivalent prophylactic human papillomavirus (types 6, 11, 16, and 18) L1 virus-like-particle vaccine against high-grade vulval and vaginal lesions: a combined analysis of three randomised clinical trials. Lancet. 2007;369(9574):1693–702.Google Scholar
Joura, EA, Giuliano, AR, Iversen, OE, et al. A 9-valent HPV vaccine against infection and intraepithelial neoplasia in women. N Engl J Med. 2015;372(8):711–23.CrossRefGoogle ScholarPubMed
Huh, WK, Joura, EA, Giuliano, AR, et al. Final efficacy, immunogenicity, and safety analyses of a nine-valent human papillomavirus vaccine in women aged 16–26 years: a randomised, double-blind trial. Lancet. 2017;390(10108):2143–59.CrossRefGoogle Scholar
Chen, YH, Gesser, R, Luxembourg, A. A seamless phase IIB/III adaptive outcome trial: design rationale and implementation challenges. Clin Trials. 2015;12(1):8490.CrossRefGoogle ScholarPubMed
Reverberi, R. The statistical analysis of immunohaematological data. Blood Transfus. 2008;6(1):3745.Google Scholar
Ravandi, F, Ritchie, EK, Sayar, H, et al. Vosaroxin plus cytarabine versus placebo plus cytarabine in patients with first relapsed or refractory acute myeloid leukaemia (VALOR): a randomised, controlled, double-blind, multinational, phase 3 study. Lancet Oncol. 2015;16(9):1025–36.CrossRefGoogle ScholarPubMed
Breems, DA, Van Putten, WL, Huijgens, PC, et al. Prognostic index for adult patients with acute myeloid leukemia in first relapse. J Clin Oncol. 2005;23(9):1969–78.CrossRefGoogle ScholarPubMed
Lancet, JE, Roboz, GJ, Cripe, LD, et al. A phase 1b/2 study of vosaroxin in combination with cytarabine in patients with relapsed or refractory acute myeloid leukemia. Haematologica. 2015;100(2):231–7.Google Scholar
Mehta, CR, Pocock, SJ. Adaptive increase in sample size when interim results are promising: a practical guide with examples. Stat Med. 2011;30(28):3267–84.Google ScholarPubMed
David, FS, Bobulsky, S, Schulz, K, Patel, N. Creating value with financially adaptive clinical trials. Nat Rev Drug Discov. 2015;14(8):523–4.CrossRefGoogle ScholarPubMed
Jones, AE, Puskarich, MA, Shapiro, NI, et al. Effect of levocarnitine vs placebo as an adjunctive treatment for septic shock: the Rapid Administration of Carnitine in Sepsis (RACE) randomized clinical trial. JAMA Netw Open. 2018;1(8):e186076.CrossRefGoogle ScholarPubMed
Calvani, M, Reda, E, Arrigoni-Martelli, E. Regulation by carnitine of myocardial fatty acid and carbohydrate metabolism under normal and pathological conditions. Basic Res Cardiol. 2000;95(2):7583.CrossRefGoogle ScholarPubMed
Krams, M, Lees, KR, Hacke, W, et al. Acute Stroke Therapy by Inhibition of Neutrophils (ASTIN): an adaptive dose-response study of UK-279,276 in acute ischemic stroke. Stroke. 2003;34(11):2543–8.CrossRefGoogle ScholarPubMed
Grieve, AP, Krams, M. ASTIN: a Bayesian adaptive dose-response trial in acute stroke. Clin Trials. 2005;2(4):340–51.Google Scholar
Jones, RL, Ravi, V, Brohl, AS, et al. Efficacy and safety of TRC105 plus pazopanib vs pazopanib alone for treatment of patients with advanced angiosarcoma: a randomized clinical trial. JAMA Oncol. 2022;8(5):740–7.CrossRefGoogle ScholarPubMed
Mehta, CR, Liu, L, Theuer, C. An adaptive population enrichment phase III trial of TRC105 and pazopanib versus pazopanib alone in patients with advanced angiosarcoma (TAPPAS trial). Ann Oncol. 2019; 30(1):103–8.Google Scholar

References

Park, JJH, Siden, E, Zoratti, MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials. 2019;20(1):572.CrossRefGoogle ScholarPubMed
Vanderbeek, AM, Bliss, JM, Yin, Z, Yap, C. Implementation of platform trials in the COVID-19 pandemic: a rapid review. Contemp Clin Trials. 2022;112:106625.CrossRefGoogle ScholarPubMed
Parmar, MK, Barthel, FM, Sydes, M, et al. Speeding up the evaluation of new agents in cancer. J Natl Cancer Inst. 2008;100(17):1204–14.CrossRefGoogle ScholarPubMed
James, ND, Sydes, MR, Clarke, NW, et al. Systemic therapy for advancing or metastatic prostate cancer (STAMPEDE): a multi-arm, multistage randomized controlled trial. BJU Int. 2009;103(4):464–9.CrossRefGoogle ScholarPubMed
Sydes, MR, Parmar, MKB, James, ND, et al. Issues in applying multi-arm multi-stage methodology to a clinical trial in prostate cancer: the MRC STAMPEDE trial. Trials. 2009;10:39.Google Scholar
National Library of Medicine. Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy (STAMPEDE). ClinicalTrials.gov Identifier NCT00268476; 22 December 2005, last updated 13 April 2022. https://clinicaltrials.gov/ct2/show/NCT00268476Google Scholar
James, ND, Sydes, MR, Clarke, NW, et al. Addition of docetaxel, zoledronic acid, or both to first-line long-term hormone therapy in prostate cancer (STAMPEDE): survival results from an adaptive, multiarm, multistage, platform randomised controlled trial. Lancet. 2016;387(10024):1163–77.Google Scholar
Mason, MD, Clarke, NW, James, ND, et al. Adding celecoxib with or without zoledronic acid for hormone-naïve prostate cancer: long-term survival results from an adaptive, multiarm, multistage, platform, randomized controlled trial. J Clin Oncol. 2017;35(14):1530.CrossRefGoogle ScholarPubMed
James, ND, de Bono, JS, Spears, MR, et al. Abiraterone for Prostate cancer not previously treated with hormone therapy. N Engl J Med. 2017;377(4):338–51.Google Scholar
Parker, CC, James, ND, Brawley, CD, et al. Radiotherapy to the primary tumour for newly diagnosed, metastatic prostate cancer (STAMPEDE): a randomised controlled phase 3 trial. Lancet. 2018;392(10162):2353–66.Google Scholar
Attard, G, Murphy, L, Clarke, NW, et al. Abiraterone acetate and prednisolone with or without enzalutamide for high-risk non-metastatic prostate cancer: a meta-analysis of primary results from two randomised controlled phase 3 trials of the STAMPEDE platform protocol. Lancet. 2022;399(10323):447–60.CrossRefGoogle ScholarPubMed
James, ND, Spears, MR, Clarke, NW, et al. Failure-free survival and radiotherapy in patients with newly diagnosed nonmetastatic prostate cancer: data from patients in the control arm of the STAMPEDE trial. JAMA Oncol. 2016;2(3):348–57.CrossRefGoogle ScholarPubMed
Redman, MW, Allegra, CJ. The master protocol concept. Semin Oncol. 2015;42(5):724–30.Google Scholar
Royston, P, Parmar, MK, Qian, W. Novel designs for multi-arm clinical trials with survival outcomes with an application in ovarian cancer. Stat Med. 2003;22(14):2239–56.CrossRefGoogle ScholarPubMed
Millen, GC, Yap, C. Adaptive trial designs: what are multiarm, multistage trials? Arch Dis Child Educ Prac. 2020;105(6):376–8.Google ScholarPubMed
Park, JJH, Harari, O, Dron, L, et al. An overview of platform trials with a checklist for clinical readers. J Clin Epidemiol. 2020;125:18.CrossRefGoogle ScholarPubMed
Angus, DC, Alexander, BM, Berry, S, et al. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov. 2019;18(10):797807.Google Scholar
Sydes, MR, James, ND, Mason, MD, et al. Flexible trial design in practice – dropping and adding arms in STAMPEDE: a multi-arm multi-stage randomised controlled trial. Trials. 2011;12(suppl. 1).Google Scholar
Sydes, MR, Parmar, MKB, Mason, MD, et al. Flexible trial design in practice – stopping arms for lack-of-benefit and adding research arms mid-trial in STAMPEDE: a multi-arm multi-stage randomized controlled trial. Trials. 2012;13:168.Google Scholar
Sweeney, CJ, Chen, YH, Carducci, M, et al. Chemohormonal therapy in metastatic hormone-sensitive prostate cancer. N Engl J Med. 2015;373(8):737–46.Google Scholar
Gravis, G, Boher, JM, Joly, F, et al. Androgen deprivation therapy (ADT) plus docetaxel versus ADT alone in metastatic non castrate prostate cancer: impact of metastatic burden and long-term survival analysis of the randomized phase 3 GETUG-AFU15 trial. Eur Urol. 2016;70(2):256–62.Google Scholar
Vale, CL, Burdett, S, Rydzewska, LHM, et al. Addition of docetaxel or bisphosphonates to standard of care in men with localised or metastatic, hormone-sensitive prostate cancer: a systematic review and meta-analyses of aggregate data. Lancet Oncol. 2016;17(2):243–56.Google Scholar
Park, JJH, Detry, MA, Murthy, S, Guyatt, G, Mills, EJ. How to use and interpret the results of a platform trial: users’ guide to the medical literature. JAMA. 2022;327(1):6774.CrossRefGoogle ScholarPubMed
Woodcock, J, LaVange, LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med. 2017;377(1):6270.Google Scholar
Park, JW, Liu, MC, Yee, D, et al. Adaptive randomization of neratinib in early breast cancer. N Engl J Med. 2016;375(1):1122.CrossRefGoogle ScholarPubMed
Rugo, HS, Olopade, OI, DeMichele, A, et al. Adaptive randomization of veliparib-carboplatin treatment in breast cancer. N Engl J Med. 2016;375(1):2334.CrossRefGoogle ScholarPubMed
Barker, AD, Sigman, CC, Kelloff, GJ, et al. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther. 2009;86(1):97100.CrossRefGoogle ScholarPubMed
Mook, S, Van’t Veer, LJ, Rutgers, EJ, Piccart-Gebhart, MJ, Cardoso, F. Individualization of therapy using Mammaprint: from development to the MINDACT trial. Cancer Genomics Proteomics. 2007;4(3):147–55.Google Scholar
Cardoso, F, Van’t Veer, L, Rutgers, E, et al. Clinical application of the 70-gene profile: the MINDACT trial. J Clin Oncol. 2008;26(5):729–35.CrossRefGoogle ScholarPubMed
National Library of Medicine. I-SPY TRIAL: Neoadjuvant and Personalized Adaptive Novel Agents to Treat Breast Cancer (I-SPY). ClinicalTrials.gov Identifier NCT01042379; 5 January 2010; last updated 10 June 2022. https://clinicaltrials.gov/ct2/show/NCT01042379.Google Scholar
Das, S, Lo, AW. Re-inventing drug development: a case study of the I-SPY 2 breast cancer clinical trials program. Contemp Clin Trials. 2017;62:168–74.Google Scholar
Chien, AJ, Tripathy, D, Albain, KS, et al. MK-2206 and standard neoadjuvant chemotherapy improves response in patients with human epidermal growth factor receptor 2–positive and/or hormone receptor–negative breast cancers in the I-SPY 2 trial. J Clin Oncol. 2020;38(10):1059.CrossRefGoogle ScholarPubMed
Nanda, R, Liu, MC, Yau, C, et al. Effect of pembrolizumab plus neoadjuvant chemotherapy on pathologic complete response in women with early-stage breast cancer: an analysis of the ongoing phase 2 adaptively randomized I-SPY2 trial. JAMA Oncol. 2020;6(5):676–84.CrossRefGoogle ScholarPubMed
Wise, J, Coombes, R. Covid-19: the inside story of the RECOVERY trial. BMJ. 2020;370:m2670.Google Scholar
The RECOVERY trial. 2020. www.recoverytrial.net/Google Scholar
Park, JJ, Mogg, R, Smith, GE, et al. How COVID-19 has fundamentally changed clinical research in global health. Lancet Glob Health. 2021;9(5):e711–e20.CrossRefGoogle ScholarPubMed
Park, JJ, Ford, N, Xavier, D, et al. Randomised trials at the level of the individual. Lancet Glob Health. 2021;9(5):e691e700.Google Scholar
Park, JJ, Grais, RF, Taljaard, M, et al. Urgently seeking efficiency and sustainability of clinical trials in global health. Lancet Glob Health. 2021;9(5):e681–e90.CrossRefGoogle ScholarPubMed
Narhi, F, Moonesinghe, SR, Shenkin, SD, et al. Implementation of corticosteroids in treatment of COVID-19 in the ISARIC WHO Clinical Characterisation Protocol UK: prospective, cohort study. Lancet Digit Health. 2022;4(4):e220–e34.Google ScholarPubMed
Recovery Collaborative Group, Horby, P, Lim, WS, Emberson, JR, et al. Dexamethasone in hospitalized patients with Covid-19. N Engl J Med. 2021;384(8):693704.Google Scholar
RC Group, Horby, P, Lim, WS, Emberson, JR, et al. Dexamethasone in hospitalized patients with Covid-19. N Engl J Med. 2021;384(8):693704.Google ScholarPubMed
NIHR. Recruiting patients for clinical trials for COVID-19 Therapeutics; 2020. www.nihr.ac.uk/documents/news/recruiting-patients-for-clinical-trials-for-covid-therapeutics.pdfGoogle Scholar
Horby, P, Lim, WS, Emberson, J, et al. Effect of dexamethasone in hospitalized patients with COVID-19: preliminary report. medRxiv. 2020:2020.06.22.20137273.Google Scholar
RC Group. Casirivimab and imdevimab in patients admitted to hospital with COVID-19 (RECOVERY): a randomised, controlled, open-label, platform trial. Lancet. 2022;399(10325):665–76.Google Scholar
RC Group. Aspirin in patients admitted to hospital with COVID-19 (RECOVERY): a randomised, controlled, open-label, platform trial. Lancet. 2022;399(10320):143–51.Google Scholar
RC Group. Colchicine in patients admitted to hospital with COVID-19 (RECOVERY): a randomised, controlled, open-label, platform trial. Lancet Respir Med. 2021;9(12):1419–26.Google Scholar
RC Group. Convalescent plasma in patients admitted to hospital with COVID-19 (RECOVERY): a randomised controlled, open-label, platform trial. Lancet. 2021;397(10289):2049–59.Google Scholar
RC Group. Tocilizumab in patients admitted to hospital with COVID-19 (RECOVERY): a randomised, controlled, open-label, platform trial. Lancet. 2021;397(10285):1637–45.Google Scholar
RC Group. Azithromycin in patients admitted to hospital with COVID-19 (RECOVERY): a randomised, controlled, open-label, platform trial. Lancet. 2021;397(10274):605–12.Google Scholar
RC Group. Lopinavir-ritonavir in patients admitted to hospital with COVID-19 (RECOVERY): a randomised, controlled, open-label, platform trial. Lancet. 2020;396(10259):1345–52.Google Scholar
RC Group, Horby, P, Mafham, M, et al. Effect of hydroxychloroquine in hospitalized patients with Covid-19. N Engl J Med. 2020;383(21):2030–40.Google Scholar
Park, JJH, Dron, L, Mills, EJ. Moving forward in clinical research with master protocols. Contemp Clin Trials. 2021;106:106438.Google Scholar
Forrest, JI, Rawat, A, Duailibe, F, et al. Resilient clinical trial infrastructure in response to the COVID-19 pandemic: lessons learned from the TOGETHER randomized platform clinical trial. Am J Trop Med Hyg. 2022;106(2):389–93.CrossRefGoogle Scholar
Reis, G, Moreira Silva, EAdS, Medeiros Silva, DC, et al. Effect of early treatment with hydroxychloroquine or lopinavir and ritonavir on risk of hospitalization among patients with COVID-19: the TOGETHER randomized clinical trial. JAMA Netw Open. 2021;4(4):e216468–e.Google Scholar
Reis, G, Moreira Silva, EAdS, Silva, DCM, et al. A multi-center, adaptive, randomized, platform trial to evaluate the effect of repurposed medicines in outpatients with early coronavirus disease 2019 (COVID-19) and high-risk for complications: the TOGETHER master trial protocol. Gates Open Res. 2021;5:117.CrossRefGoogle Scholar
Reis, G, Silva, E, Silva, DCM, et al. Effect of early treatment with ivermectin among patients with Covid-19. N Engl J Med. 2022.CrossRefGoogle Scholar
Reis, G, Dos Santos Moreira-Silva, EA, Silva, DCM, et al. Effect of early treatment with fluvoxamine on risk of emergency care and hospitalisation among patients with COVID-19: the TOGETHER randomised, platform clinical trial. Lancet Glob Health. 2022;10(1):e42e51.CrossRefGoogle ScholarPubMed
Reis, G, Silva, EAdSM, Silva, DCM, et al. Effect of early treatment with metformin on risk of emergency care and hospitalization among patients with COVID-19: the TOGETHER randomized platform clinical trial. Lancet Regional Health-Americas. 2022;6:100142.Google Scholar
Lee, TC, Vigod, S, Bortolussi-Courval, E, et al. Fluvoxamine for outpatient management of COVID-19 to prevent hospitalization: a systematic review and meta-analysis. JAMA Netw Open. 2022; 5(4):e226269.Google Scholar

References

Connell, CM, Doherty, GJ. Activating HER2 mutations as emerging targets in multiple solid cancers. ESMO Open. 2017;2(5):e000279.Google Scholar
Verma, S, Miles, D, Gianni, L, et al. Trastuzumab emtansine for HER2-positive advanced breast cancer. N Engl J Med. 2012;367(19):1783–91.CrossRefGoogle ScholarPubMed
Amiri-Kordestani, L, Blumenthal, GM, Xu, QC, et al. FDA approval: ado-trastuzumab emtansine for the treatment of patients with HER2-positive metastatic breast cancer. Clin Cancer Res. 2014;20(17):4436–41.Google ScholarPubMed
Li, BT, Shen, R, Buonocore, D, et al. Ado-trastuzumab emtansine for patients with HER2-mutant lung cancers: results from a phase II basket trial. J Clin Oncol. 2018;36(24):2532–7.Google Scholar
Li, BT, Makker, V, Buonocore, DJ, et al. A multi-histology basket trial of ado-trastuzumab emtansine in patients with HER2 amplified cancers. J Clin Oncol. 2018;36(15_suppl):2502.CrossRefGoogle Scholar
Eisenhauer, EA, Therasse, P, Bogaerts, J, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009;45(2):228–47.Google Scholar
Simon, R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials. 1989;10(1):110.Google Scholar
Park, JJ, Harari, O, Dron, L, Mills, EJ, Thorlund, K. Effects of biomarker diagnostic accuracy on biomarker-guided phase 2 trials. Contemp Clin Trials Commun. 2019;15:100396.Google Scholar
Conley, BA, Doroshow, JH. Molecular analysis for therapy choice: NCI MATCH. Semin Oncol. 2014;41(3):297–9.Google Scholar
Mullard, A. NCI-MATCH trial pushes cancer umbrella trial paradigm. Nat Rev Drug Discov. 2015;14(8):513–5.Google ScholarPubMed
Chen, A, Conley, B, Hamilton, S, et al. NCI-Molecular Analysis for Therapy Choice (NCI-MATCH) trial: a novel public-private partnership. Eur J Cancer. 2016;69:S137.Google Scholar
Moore, KN, Mannel, RS. Is the NCI MATCH trial a match for gynecologic oncology? Gynecol Oncol. 2016;140(1):161–6.Google Scholar
Abrams, J, Conley, B, Mooney, M, et al. National Cancer Institute’s Precision Medicine Initiatives for the new National Clinical Trials Network. Am Soc Clin Oncol Educ Book. 2014:71–6.Google Scholar
Barroilhet, L, Matulonis, U. The NCI-MATCH trial and precision medicine in gynecologic cancers. Gynecol Oncol. 2018;148(3):585–90.Google Scholar
Chae, YK, Hong, F, Vaklavas, C, et al. Phase II study of AZD4547 in patients with tumors harboring aberrations in the FGFR pathway: results from the NCI-MATCH trial (EAY131) subprotocol W. J Clin Oncol. 2020;38(21):2407–17.Google Scholar
Mansfield, AS, Wei, Z, Mehra, R, et al. Crizotinib in patients with tumors harboring ALK or ROS1 rearrangements in the NCI-MATCH trial. NPJ Precis Oncol. 2022;6(1):13.Google Scholar
Van Cutsem, E, Bang, YJ, Mansoor, W, et al. A randomized, open-label study of the efficacy and safety of AZD4547 monotherapy versus paclitaxel for the treatment of advanced gastric adenocarcinoma with FGFR2 polysomy or gene amplification. Ann Oncol. 2017;28(6):1316–24.Google Scholar
Aggarwal, C, Redman, MW, Lara, PN, Jr., et al. SWOG S1400D (NCT02965378), a phase II study of the fibroblast growth factor receptor inhibitor AZD4547 in previously treated patients with fibroblast growth factor pathway-activated stage IV squamous cell lung cancer (Lung-MAP Substudy). J Thorac Oncol. 2019;14(10):1847–52.CrossRefGoogle ScholarPubMed
Aggarwal, C, Redman, MW, Lara, P, et al. Phase II study of the FGFR inhibitor AZD4547 in previously treated patients with FGF pathway-activated stage IV squamous cell lung cancer (SqNSCLC): LUNG-MAP sub-study SWOG S1400D. J Clin Oncol. 2017;35(15 suppl).Google Scholar
Aggarwal, C, Redman, MW, Lara, PN, Jr et al. SWOG S1400D (NCT02965378), a phase II study of the fibroblast growth factor receptor inhibitor AZD4547 in previously treated patients with fibroblast growth factor pathway–activated stage IV squamous cell lung cancer (Lung-MAP substudy). J Thorac Oncol. 2010;14(10):18471852.Google Scholar
Lih, CJ, Takebe, N. Considerations of developing an NGS assay for clinical applications in precision oncology: The NCI-MATCH NGS assay experience. Curr Probl Cancer. 2017;41(3):201–11.CrossRefGoogle ScholarPubMed
Kummar, S, Williams, M, Lih, C-J, et al. NCI mpact: National Cancer Institute molecular profiling-based assignment of cancer therapy. J Clin Oncol. 2014;32(15).Google Scholar
Chen, AP, Kummar, S, Moore, N, et al. Molecular Profiling-Based Assignment of Cancer Therapy (NCI-MPACT): a randomized multicenter phase II trial. JCO Precis Oncol. 2021;5.Google Scholar
Turner, N, Bye, H, Kernaghan, S, et al. Abstract OT1-06-03: The plasmaMATCH trial: A multiple parallel cohort, open-label, multi-centre phase II clinical trial of ctDNA screening to direct targeted therapies in patients with advanced breast cancer (CRUK/15/010). Cancer Res. 2018;78(4 Supplement):OT1-06-3-OT1–3.Google Scholar
Cancer Research UK. A trial using a blood test to find certain gene changes and decide treatment for advanced breast cancer (plasmaMATCH). 2019. www.cancerresearchuk.org/about-cancer/find-a-clinical-trial/a-trial-using-a-blood-test-to-find-certain-gene-changes-and-decide-treatment-for-advanced-breast#undefinedGoogle Scholar
Kingston, B, Cutts, RJ, Bye, H, et al. Genomic profile of advanced breast cancer in circulating tumour DNA. Nat Commun. 2021;12(1):2423.Google Scholar
Turner, NC, Kingston, B, Kilburn, LS, et al. Circulating tumour DNA analysis to direct therapy in advanced breast cancer (plasmaMATCH): a multicentre, multicohort, phase 2a, platform trial. Lancet Oncol. 2020;21(10):1296–308.CrossRefGoogle ScholarPubMed
Covens, AL, Filiaci, V, Gersell, D, et al. Phase II study of fulvestrant in recurrent/metastatic endometrial carcinoma: a Gynecologic Oncology Group study. Gynecol Oncol. 2011;120(2):185–8.Google Scholar
Rabindran, SK, Discafani, CM, Rosfjord, EC, et al. Antitumor activity of HKI-272, an orally active, irreversible inhibitor of the HER-2 tyrosine kinase. Cancer Res. 2004;64(11):3958–65.Google Scholar
Fong, PC, Boss, DS, Yap, TA, et al. Inhibition of poly(ADP-ribose) polymerase in tumors from BRCA mutation carriers. N Engl J Med. 2009;361(2):123–34.Google Scholar
Govindan, R, Mandrekar, SJ, Gerber, DE, Ox, , et al. ALCHEMIST trials: a golden opportunity to transform outcomes in early-stage non-small cell lung cancer. Clin Cancer Res. 2015;21(24):5439–44.Google Scholar
Kehl, KL, Zahrieh, D, Yang, P, et al. Rates of guideline-concordant surgery and adjuvant chemotherapy among patients with early-stage lung cancer in the US ALCHEMIST study (Alliance A151216). JAMA Oncol. 2022;8(5):717–28.CrossRefGoogle ScholarPubMed
Chaft, JE, Dahlberg, SE, Khullar, OV, et al. EA5142 adjuvant nivolumab in resected lung cancers (ANVIL). J Clin Oncol. 2018;35(15).Google Scholar
Sands, J, Mandrekar, SJ, Oxnard, GR, K, et al. ALCHEMIST: Adjuvant targeted therapy or immunotherapy for high-risk resected NSCLC. J Clin Oncol. 2020;28(15 suppl).Google Scholar
Ferrarotto, R, Redman, MW, Gandara, DR, Herbst, RS, Papadimitrakopoulou, VA. Lung-MAP – framework, overview, and design principles. Chin Clin Oncol. 2015;4(3):36.Google Scholar
SWOG Cancer Research Network. A Master Protocol to Evaluate Biomarker-Driven Therapies and Immunotherapies in Previously-Treated Non-Small Cell Lung Cancer (Lung-MAP Screening Study). 2019. www.swog.org/clinical-trials/lungmapGoogle Scholar
Papadimitrakopoulou, V, Redman, M, Borghaei, H, et al. 83OA phase II study of durvalumab (MEDI4736) for previously treated patients with stage IV squamous NSCLC (SqNSCLC): Lung-MAP Sub-study SWOG S1400A. Ann Oncol. 2017;28(suppl_2).CrossRefGoogle Scholar
Wade, JL, Langer, CJ, Redman, M, et al. A phase II study of GDC-0032 (taselisib) for previously treated PI3 K positive patients with stage IV squamous cell lung cancer (SqNSCLC): LUNG-MAP sub-study SWOG S1400B. J Clin Oncol. 2017;35(15).Google Scholar
Edelman, MJ, Redman, MW, Albain, KS, et al. A phase II study of palbociclib (P) for previously treated cell cycle gene alteration positive patients (pts) with stage IV squamous cell lung cancer (SCC): Lung-MAP sub-study SWOG S1400C. J Clin Oncol. 2017;35(15).Google Scholar
Borghaei, H, Redman, MW, Kelly, K, et al. SWOG S1400A (NCT02154490): a phase II study of durvalumab for patients with previously treated stage IV or recurrent squamous cell lung cancer (Lung-MAP sub-study). Clin Lung Cancer. 2021;22(3):178–86.Google Scholar
Langer, CJ, Redman, MW, Wade, JL, 3rd, et al. SWOG S1400B (NCT02785913), a phase ii study of GDC-0032 (taselisib) for previously treated PI3 K-positive patients with stage IV squamous cell lung cancer (Lung-MAP sub-study). J Thorac Oncol. 2019;14(10):1839–46.Google Scholar
Edelman, MJ, Redman, MW, Albain, KS, et al. SWOG S1400C (NCT02154490) – a phase II study of palbociclib for previously treated cell cycle gene alteration-positive patients with stage IV squamous cell lung cancer (Lung-MAP substudy). J Thorac Oncol. 2019;14(10):1853–9.CrossRefGoogle ScholarPubMed
Leighl, NB, Redman, MW, Rizvi, N, et al. Phase II study of durvalumab plus tremelimumab as therapy for patients with previously treated anti-PD-1/PD-L1 resistant stage IV squamous cell lung cancer (Lung-MAP substudy S1400F, NCT03373760). J Immunother Cancer. 2021;9(8).Google Scholar
Owonikoko, TK, Redman, MW, Byers, LA, et al. Phase 2 study of talazoparib in patients with homologous recombination repair-deficient squamous cell lung cancer: Lung-MAP substudy S1400G. Clin Lung Cancer. 2021;22(3):187–94 e1.Google Scholar
Gettinger, SN, Redman, MW, Bazhenova, L, et al. Nivolumab plus ipilimumab vs nivolumab for previously treated patients with stage IV squamous cell lung cancer: the Lung-MAP S1400I phase 3 randomized clinical trial. JAMA Oncol. 2021;7(9):1368–77.Google Scholar
Waqar, SN, Redman, MW, Arnold, SM, et al. A phase II study of telisotuzumab vedotin in patients with c-MET-positive stage IV or recurrent squamous cell lung cancer (LUNG-MAP Sub-study S1400 K, NCT03574753). Clin Lung Cancer. 2021;22(3):170–7.Google Scholar
Roth, JA, Trivedi, MS, Gray, SW, et al. Patient knowledge and expectations about return of genomic results in a biomarker-driven master protocol trial (SWOG S1400GEN). JCO Oncology Pract. 2021;17(11):e1821–e9.Google Scholar
Redman, MW, Papadimitrakopoulou, VA, Minichiello, K, et al. Biomarker-driven therapies for previously treated squamous non-small-cell lung cancer (Lung-MAP SWOG S1400): a biomarker-driven master protocol. Lancet Oncol. 2020;21(12):1589–601.Google Scholar

References

Friedman, LM, Furberg, C, DeMets, DL, Reboussin, D, Granger, CB. Fundamentals of Clinical Trials. Springer; 2015.CrossRefGoogle Scholar
Chan, AW, Tetzlaff, JM, Altman, DG, et al. SPIRIT 2013 statement: defining standard protocol items for clinical trials. Ann Intern Med. 2013;158(3):200207.Google Scholar
Schulz, KF, Altman, DG, Moher, D, Group C. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. Trials. 2010;11:32.Google Scholar
Schulz, KF, Altman, DG, Moher, D, Group C. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c332.CrossRefGoogle ScholarPubMed
Sterne, JAC, Savovic, J, Page, MJ, et al. RoB 2: a revised tool for assessing risk of bias in randomised trials. BMJ. 2019;366:l4898.CrossRefGoogle ScholarPubMed
Park, JJH, Dron, L, Mills, EJ. Moving forward in clinical research with master protocols. Contemp Clin Trials. 2021;106:106438.Google Scholar
Bhatt, DL, Mehta, C. Adaptive designs for clinical trials. N Engl J Med. 2016;375(1):6574.Google Scholar
Van Norman, GA. Phase II trials in drug development and adaptive trial design. JACC Basic Transl Sci. 2019;4(3):428–37.Google ScholarPubMed
Dimairo, M, Pallmann, P, Wason, J, et al. The adaptive designs CONSORT extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design. Trials. 2020; 21(1):528.Google Scholar
Dimairo, M, Pallmann, P, Wason, J, et al. The Adaptive designs CONSORT Extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design. BMJ. 2020;369:m115.Google Scholar
Detry, MA, Lewis, RJ, Broglio, KR, et al. Standards for the design, conduct, and evaluation of adaptive randomized clinical trials. Patient-Centered Outcomes Research Institute (PCORI), 2012.Google Scholar
United States Department of Health and Human Services, Food and Drug Administration. Adaptive Designs for Clinical Trials of Drugs and Biologics. Guidance for Industry. Center for Biologics Evaluation and Research (CBER). 2019.Google Scholar
Sydes, MR, Parmar, MK, James, ND, et al. Issues in applying multi-arm multi-stage methodology to a clinical trial in prostate cancer: the MRC STAMPEDE trial. Trials. 2009;10:39.Google Scholar
Park, JJH, Harari, O, Dron, L, et al. An overview of platform trials with a checklist for clinical readers. J Clin Epidemiol. 2020;125:18.Google Scholar
Mayer, C, Perevozskaya, I, Leonov, S, et al. Simulation practices for adaptive trial designs in drug and device development. Statistics in Biopharmaceutical Research. 2019;11(4):325–35.Google Scholar
Thorlund, K, Haggstrom, J, Park, JJ, Mills, EJ. Key design considerations for adaptive clinical trials: a primer for clinicians. BMJ. 2018;360:k698.Google Scholar
He, W, Kuznetsova, OM, Harmer, M, et al. Practical considerations and strategies for executing adaptive clinical trials. DIJ. 2012;46(2):160–74.Google Scholar
Fisher, MR, Roecker, EB, DeMets, DL. The role of an independent statistical analysis center in the institutes of health model. DIJ. 2001;35(1):115–29.Google Scholar
Juszczak, E, Altman, DG, Hopewell, S, Schulz, K. Reporting of multi-arm parallel-group randomized trials: extension of the CONSORT 2010 statement. JAMA. 2019;321(16):1610–20.Google Scholar
Logullo, P, MacCarthy, A, Kirtley, S, Collins, GS. Reporting guideline checklists are not quality evaluation forms: they are guidance for writing. Health Sci Rep. 2020;3(2).Google Scholar
Park, JJH, Siden, E, Zoratti, MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials. 2019; 20(1):572.Google Scholar
Siden, EG, Park, JJ, Zoratti, MJ, et al. Reporting of master protocols towards a standardized approach: a systematic review. Contemp Clin Trials Commun. 2019;15:100406.Google Scholar
Schiavone, F, Bathia, R, Letchemanan, K, et al. This is a platform alteration: a trial management perspective on the operational aspects of adaptive and platform and umbrella protocols. Trials. 2019; 20(1):264.Google Scholar
Morrell, L, Hordern, J, Brown, L, et al. Mind the gap? The platform trial as a working environment. Trials. 2019; 20(1):16.Google Scholar
Hague, D, Townsend, S, Masters, L, et al. Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons. Trials. 2019; 20(1):116.CrossRefGoogle ScholarPubMed
Park, JJH, Hsu, G, Siden, EG, Thorlund, K, Mills, EJ. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin. 2020;70(2):125–37.Google Scholar
Antoniou, M, Kolamunnage-Dona, R, Wason, J, et al. Biomarker-guided trials: challenges in practice. Contemp Clin Trials Commun. 2019;16:100493.Google Scholar
Park, JJH, Detry, MA, Murthy, S, Guyatt, G, Mills, EJ. How to use and interpret the results of a platform trial: users’ guide to the medical literature. JAMA. 2022;327(1):6774.Google Scholar
Orkin, AM, Gill, PJ, Ghersi, D, et al. Guidelines for reporting trial protocols and completed trials modified due to the COVID-19 pandemic and other extenuating circumstances: the CONSERVE 2021 statement. JAMA. 2021;326(3):257–65.Google Scholar
Angus, DC, Derde, L, Al-Beidh, F, et al. Effect of hydrocortisone on mortality and organ support in patients with severe COVID-19: The REMAP-CAP COVID-19 corticosteroid domain randomized clinical trial. JAMA. 2020;324(13):1317–29.Google ScholarPubMed
Recovery Collaborative Group, Horby, P, Lim, WS, Emberson, JR, et al. Dexamethasone in hospitalized patients with Covid-19. N Engl J Med. 2021;384(8):693704.Google Scholar

Save book to Kindle

To save this book to your Kindle, first ensure coreplatform@cambridge.org is added to your Approved Personal Document E-mail List under your Personal Document Settings on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part of your Kindle email address below. Find out more about saving to your Kindle.

Note you can select to save to either the @free.kindle.com or @kindle.com variations. ‘@free.kindle.com’ emails are free but can only be saved to your device when it is connected to wi-fi. ‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.

Find out more about the Kindle Personal Document Service.

Available formats
×

Save book to Dropbox

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Dropbox.

Available formats
×

Save book to Google Drive

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Google Drive.

Available formats
×