Epub 2022 Dec 6. Why Should I Register and Submit Results? Lastly, this study focuses on right implementation for the study design and sample size calculation. Diagnosis and treatment patterns for patients with leptomeningeal metastasis from solid tumors across Europe. Naxitamab and GM-CSF will be given for at least 8 cycles. Table 1 shows the summary of the included studies for Phase 2 single-arm two-stage clinical trials in glioblastoma. Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design. Phase II study of Dovitinib in recurrent glioblastoma. 2012;110(1):1118. 2004;23(4):5619. Bookshelf Disclaimer, National Library of Medicine Page MJ, et al. Finkelstein DM, Muzikansky A, Schoenfeld DA. Federal government websites often end in .gov or .mil. Bookshelf Secondary objectives included toxicity, response rate and time to intracranial or extracranial disease progression. [56] proposed modified PFS (mPFS) for immune-oncology trials. Phase 2 trial of hypoxia activated evofosfamide (TH302) for treatment of recurrent bevacizumab-refractory glioblastoma. Fortunately, several reviews present that PFS6 and OS are generally strongly associated in glioblastoma trials, but not between RR and OS [54]. Epub 2020 Sep 1. 2021;13(15):3750. However, it is important that efficient and valid study designs be applied successfully and adequately to reach the aims of phase 2 oncology trials. 2022;169:107420. Shi YK, Hong XN, Yang JL, Xu W, Huang HQ, Xiao XB, Zhu J, Zhou DB, Han XH, Wu JQ, Zhang MZ, Jin J, Ke XY, Li W, Wu DP, Yang SM, Du X, Jia YQ, Liu AC, Liu DH, Shen ZX, Zhang LS, James L, Hellriegel E. Chin Med J (Engl). Funding: Now our interest turns to how we can choose type I and II error rates for clinical trials? More than three quarter articles (n=22, 76%) provided all related information of type I and II errors (\(\alpha , \beta\)) and unacceptable and acceptable response rates (\({p}_{0}, {p}_{1}\)). The site is secure. government site. J Neurooncol. Ishida T, Fujiwara H, Nosaka K, Taira N, Abe Y, Imaizumi Y, Moriuchi Y, Jo T, Ishizawa K, Tobinai K, Tsukasaki K, Ito S, Yoshimitsu M, Otsuka M, Ogura M, Midorikawa S, Ruiz W, Ohtsu T. J Clin Oncol. Your comment will be reviewed and published at the journal's discretion. Further investigations are needed to identify which patients with LMD can benefit from pembrolizumab. FOIA The outcomes of simulation are depicted in Fig. Wonsuk Yoo. Phase 2 study of bosutinib, a Src inhibitor, in adults with recurrent glioblastoma. For good study design with phase II trials, investigators should maintain low level of type I and II errors. 2013;10(5):72034. J Neurooncol. 1. official website and that any information you provide is encrypted An increasing fraction of patients with metastatic cancer develop leptomeningeal dissemination of disease (LMD), and survival is dismal1-3. and transmitted securely. Ivy Brain Tumor Center, Department of Translational Neuroscience, Barrow Neurological Institute, Phoenix, AZ, 85013, USA, Karmanos Cancer Institute, Department of Oncology, School of Medicine, Wayne State University, Detroit, MI, 48201, USA, Department of Radiation Oncology, Barrow Neurological Institute, Phoenix, AZ, 85013, USA, You can also search for this author in Epub 2017 May 24. PMC Careers. The evaluation of PFS6 currently relies solely on a point estimation after dichotomization of PFS6 into a binary endpoint, which may cause issues discussed previously. Protracted low doses of temozolomide for the treatment of patients with recurrent glioblastoma: a phase II study. safety; single-stage design; tolerability; toxicity; two-stage design. Jung SH, et al. Contemp Clin Trials. Federal government websites often end in .gov or .mil. Comput Stat Data Anal. A phase II single-arm study of irinotecan in combination with temozolomide (TEMIRI) in children with newly diagnosed high grade glioma: a joint ITCC and SIOPE-brain tumour study. Such an unexpected situation cannot be appropriately handled with the current system of two-stage designs because current designs require to prespecify the design information like sample size for each stage and stopping rules in the study protocol. We conducted a single-arm, phase 2 study of pembrolizumab in patients with solid tumor malignancies and LMD (NCT02886585). Talk with your doctor and family members or friends about deciding to join a study. Childs Nerv Syst. CAS Phase II study of ipilimumab and nivolumab in leptomeningeal carcinomatosis. 2015;17(7):9928. Clinical Trial NCT04962126; Frontline Treatment of Follicular Lymphoma With AtezolizUmab and Obinutuzumab With and Without RadiOtherapy (FLUORO) January 13, 2023 updated by: Olivia Newton-John Cancer Research Institute. Safety and efficacy of decitabine in combination with temozolomide in metastatic melanoma: a phase I/II study and pharmacokinetic analysis. Article Wu X, Wu C, Liu F, Zhou H, Chen C. A generalized framework of optimal two-stage designs for exploratory basket trials. The .gov means its official. Patients received valemetostat 200 mg/day until progressive disease or unacceptable toxicity. Schmidt R, Faldum A, Kwiecien R. Adaptive designs for the one-sample log-rank test. sharing sensitive information, make sure youre on a federal eCollection 2021. Due to economical and ethical reasons, the two-stage designs have been widely used for Phase 2 single-arm trials in oncology because the designs allow us to stop the trial early if the proposed treatment is likely to be ineffective. Therapeutic Antibodies in Cancer Treatment in the UK. Patients will receive up to 18 IT cycles after enrollment. eCollection 2020 Dec. BMC Med Res Methodol. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. GBM: glioblastoma, PFS6: progression-free survival at 6months, Key info: key input parameters for two-stage design, Right SS: did two-stage sample size calculation be appropriately implanted? An official website of the United States government. Eltarhoni K, Kamel F, Ihebunezie K, Nisar P, Soloviev M. Int J Mol Sci. This multicenter phase 2 trial (NCT04102150; https://clinicaltrials.gov/ct2/show/NCT04102150; DS3201-A-J201) enrolled patients with R/R aggressive ATL (acute, lymphoma, unfavorable chronic type). -, Ongerboer de Visser, B. W. et al. Breslow NE. BMJ. 2021 Jun;22(6):741-742. doi: 10.1016/S1470-2045(21)00191-1. 133, 419427 (2017). Clin Cancer Res. Among examined 29 trials, 12 trials (41%) appropriately provided key input parameters and sample size results from two-stage design implementation. HHS Vulnerability Disclosure, Help 2016;43(1):138. FOIA Bethesda, MD 20894, Web Policies Loi S, Giobbie-Hurder A, Gombos A, Bachelot T, Hui R, Curigliano G, Campone M, Biganzoli L, Bonnefoi H, Jerusalem G, Bartsch R, Rabaglio-Poretti M, Kammler R, Maibach R, Smyth MJ, Di Leo A, Colleoni M, Viale G, Regan MM, Andr F; International Breast Cancer Study Group and the Breast International Group. Simon's two-stage designs are frequently used in phase II single-arm trials for efficacy studies. Eastern Cooperative Oncology Group. Stat Med. There has been the need for new two-stage designs that allow flexible modification of design parameters under the control of the Type 1 error, which is called adaptive design methods to perform arbitrary design modification under the control of the Type 1 error rate. Muhic A, et al. 2016 Dec;34(34):4086-4093. doi: 10.1200/JCO.2016.67.7732. Disclaimer, National Library of Medicine 2013;33(4):165760. The survival endpoint was introduced as Bayesian extension of Simons two-stage design and R package BayesDesign [57]. Kwak M, Jung S-H. Invest New Drugs. Phase II trial of ponatinib in patients with bevacizumab-refractory glioblastoma. Cancer Med. Hyun JW, Kim Y, Kim KH, Kim SH, Park EY, Youn JH, Yoo H, Gwak HS, Kim HJ. Active disease despite previous aggressive multi-drug chemotherapy, defined as one of the following: The patients must have one of the following (locally assessed) obtained within 3 weeks prior to enrollment and at least 10 calendar days after end of any prior anti-cancer treatment: Unacceptable hematological status at screening, defined as one of the following: Unacceptable liver function at screening, defined as one of the following: To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor. 2011;103(3):491501. https://doi.org/10.1186/s12874-022-01810-7, DOI: https://doi.org/10.1186/s12874-022-01810-7. Even though 12 trials successfully implemented and reported the key input and output parameters for two-stage design sample size calculation, only 3 trials (10%) provided the references of the historical control rates for their trials. The discrete conditional error based adaptive design method requires to recalculate the probability of rejection region using the results from the first stage. The study met the primary endpoint, as 12 of 20 (OS3, 0.60; 90% confidence interval, 0.39-0.78) patients were alive at 3 months after enrollment. Before official website and that any information you provide is encrypted PRISMA 2020 explanation and elaboration: updated guidance and exemplars for reporting systematic reviews. The PFS at 6months (PFS6) is the most widely used endpoint in glioblastoma trials because of reflection of the rate of cases of durable disease control [58]. Unfortunately, most trials (n=20, 69%) failed to provide the references on the historical control rates. 2013;111(3):34753. TEAEs that occurred in 20% of patients included thrombocytopenia, anemia, alopecia, dysgeusia, neutropenia, lymphopenia, leukopenia, decreased appetite, and pyrexia. Please enable it to take advantage of the complete set of features! Suh CH, et al. The unacceptable historical control rate should be a maximum rate while the acceptable expected treatment rate should be a minimum rate in order not to be overly optimistic for the Phase 2 clinical trials. Please remove one or more studies before adding more. J Clin Oncol. Eur J Cancer. PMC In addition, the several trials provided wrong results from sample size calculation even if they reported all related key information for two-stage design implementation (not shown in table). Clipboard, Search History, and several other advanced features are temporarily unavailable. Transparency comes from providing information on historical control rates borrowed from the literature as well as expected response rates from the experimental agent. Diffuse Large B-Cell Lymphoma (DLBCL): Early Patient Management and Emerging Treatment Options. Its interesting to see that most trials without mentioning specific design types (like Simons optimal or minimax, Gehan, Fleming, or admissible designs) failed to provide one or more than one key information for the implementation results of two-stage design sample size calculation. Patterns single arm phase 2 trial patients with recurrent glioblastoma Now our interest turns to how we can type! B-Cell Lymphoma ( DLBCL ): Early Patient Management and Emerging treatment Options a, Kwiecien R. Adaptive for! Metastatic melanoma: a phase II study conditional error based Adaptive design method requires to the! Until progressive disease or unacceptable toxicity on a federal eCollection 2021 join a study Jun ; (... Lymphoma ( DLBCL ): Early Patient Management and Emerging treatment Options 29 trials, investigators should maintain low of! 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