Here, we performed a complete systematic review on the phase 2 single-arm two-stage trials in glioblastoma to evaluate the appropriate application of the two-stage designs. For general information, Learn About Clinical Studies. The optimal design usually recommended over the minimax design because the former usually shows the smaller expected sample size [12]. 11, 561569 (1993). 2021;13(3):28694. Except for three clinical trials that didnt provide the methods used, almost all articles were Simons two-stage designs (n=23, 90%). Due to duplicates (n=10), 71 articles were eligible to assess. 2015;17(8):110613. Evaluation of two-stage designs of Phase 2 single-arm trials in glioblastoma: a systematic review. volume22, Articlenumber:327 (2022) For instance, Silvani et al. Page MJ, et al. A phase 2 study of the first imipridone ONC201, a selective DRD2 antagonist for oncology, administered every three weeks in recurrent glioblastoma. Murgo AJ, et al. 2001 May;32(2):155-62. doi: 10.1016/s0169-5002(00)00220-8. A treatment cycle is 21 days. and transmitted securely. Multicenter Phase II Study of Lenalidomide in Relapsed or Recurrent Adult T-Cell Leukemia/Lymphoma: ATLL-002. 2012;11(3):2419. We study the relationship between a two-stage design with a safety endpoint and an efficacy endpoint and use simulation studies to ascertain their properties. Neuro Oncol. Paclitaxel added to the cisplatin/etoposide regimen in extensive-stage small cell lung cancer -- the use of complete response rate as the primary endpoint in phase II trials. Twenty-five patients (median age, 69.0) with a median of 3 prior lines of therapy were enrolled; 24 had prior mogamulizumab treatment. Intraventricular methotrexate therapy of leptomeningeal metastasis from breast carcinoma. 8600 Rockville Pike Banerjee A, Tsiatis AA. 2019;37(15_suppl):1820618206. Finkelstein DM, Muzikansky A, Schoenfeld DA. Secondary objectives included toxicity, response rate and time to intracranial or extracranial disease progression. As output results, the two-stage designs (Optimal, Minimax, and Admissible designs) produce following key outputs of the number of patients (\({n}_{1}\) and \({n}_{2}\)) for stage 1 and both stages and the rejection numbers (r and \({r}_{1}\)) for both stage 1 and both stages respectively. Vanderbeek AM, et al. Nat Commun. A total of 29 articles were included into the review of Phase 2 two-stage trials in glioblastoma. Together they form a unique fingerprint. This popularity is because the two-stage design in Phase 2 single-arm trials allows for early trial termination for ineffective experimental therapies (i.e., futility). Oncotarget. The primary endpoint was met with a centrally reviewed ORR of 48.0% (90% CI, 30.5% to 65.9%), including 5 complete and 7 partial remissions. A better way to avoid the potential issues is to use a hazard ratio (HR). Phase II trial of ponatinib in patients with bevacizumab-refractory glioblastoma. Multicenter, single arm, phase II trial on the efficacy of ortataxel in recurrent glioblastoma. The systematic review followed the guidelines of the Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA)-statement (http://www.prisma-statement.org) [14]. Blood 2022; blood.2022016862. Therefore, it is highly important to provide key information about input and output parameters and detail information on the choice of historical control rates based on the reference and the rational reason on the expected target response rate based on previous studies. We conducted a single-arm, Bookshelf 2022;28(4):594602. This site needs JavaScript to work properly. Biometrics. BMC Med Res Methodol. An armof a clinical trial is a group of patients receiving a specific treatment (or no treatment). WebIn the traditional study design of a single-arm phase II cancer clinical trial, the one-sample log-rank test has been frequently used. 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. Neuro Oncol. The outcomes of simulation are depicted in Fig. For the trial, 70 children aged 618 years old with peanut allergies received 12-hour boiled peanuts for 12 weeks, 2-hour boiled peanuts for 20 weeks, and roasted peanuts for 20 weeks, to a target maintenance dose of 12 roasted peanuts daily. Epub 2022 Aug 10. J Neurooncol. Survival gain in glioblastoma patients treated with dendritic cell immunotherapy is associated with increased NK but not CD8(+) T cell activation in the presence of adjuvant temozolomide. Using a 3+3 design, 13 patients with advanced cancer were enrolled and 12 were dose-limiting toxicity (DLT) evaluable. Accessibility Bookshelf Fangusaro J, et al. 8600 Rockville Pike First, if expected sample size of minimax design is close to that of optimal design, the minimax design might be a good option over the optimal design. End of study was approximately 8 1 weeks (57 7 days) after the first drug administration. J Clin Oncol. 2021;372:n160. WebMethods: In this open-label, single-arm, single-center, exploratory phase II study, 10 patients (20 eyes) received EG-Mirotin once a day (3 mg/1.5 mL sterile saline) for 5 days The adaptive design allows the incorporation of interim results to adjust the second stage designs under still controlling the Type 1 error rate and may provide economic benefit by reducing the waste of resources (Fig. Suppose a two-stage design with a type I error no larger than \({\alpha }^{*}\) and a power no smaller than (1- \({\beta }^{*}\)) for given (\({p}_{0}\), \({p}_{1}\)). Copyright 2020 Friends of Cancer Research. Before To improve test power, we proposed a new weighted one-sample log-rank test and provided a sample size calculation formula for designing trials. 18 studies used PFS6 as their primary endpoint while others include ORR (n=8) and other (n=3). Careers. Optimized image-based surrogate endpoints in targeted therapies for glioblastoma: a systematic review and meta-analysis of phase iii randomized controlled trials. The site is secure. Brastianos PK, Lee EQ, Cohen JV, Tolaney SM, Lin NU, Wang N, Chukwueke U, White MD, Nayyar N, Kim A, Alvarez-Breckenridge C, Krop I, Mahar MK, Bertalan MS, Shaw B, Mora JL, Goss N, Subramanian M, Nayak L, Dietrich J, Forst DA, Nahed BV, Batchelor TT, Shih HA, Gerstner ER, Moy B, Lawrence D, Giobbie-Hurder A, Carter SL, Oh K, Cahill DP, Sullivan RJ. 2022 Nov;82(16):1621-1627. doi: 10.1007/s40265-022-01800-5. Lastly, this study focuses on right implementation for the study design and sample size calculation. The survival endpoint was introduced as Bayesian extension of Simons two-stage design and R package BayesDesign [57]. FOIA 1R01CA227156-01/U.S. After excluding 42 articles due to randomized trials (n=7), single-stage designs (n=5), abstracts (n=3), and inadequate information (n=27), we had 29 studies included in review. Department of Health & Human Services | NIH | National Cancer Institute (NCI)/International, 5R21CA220253-02/U.S. Cookies policy. Hematology Am Soc Hematol Educ Program. Copyright 2022 American Society of Hematology. Hu J, et al. 1990;9(3):21528. Furthermore, only nine studies (31%) provided the references of historical control rates and explanation of how they chose the rates, while most studies (n=20) did not provide the reference of historical control rates and the explanation of how they chose the historical and expected response rates for their study therapeutic drugs. Patients pretreated with mogamulizumab had an ORR of 45.8% (4 complete and 7 partial remissions). Designing phase 0 cancer clinical trials. Please check for further notifications by email. First, it is usually difficult to stop patient recruitment exactly when the number of patients for the interim or final analysis is achieved, which might result to over- or under-running. 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. Now our interest turns to how we can choose type I and II error rates for clinical trials? 2004;60(2):48290. In the classical approach, further 22 patients should be recruited for the second stage although only 3 (13.6%) further patients without progression after 4months are required to demonstrate efficacy. 2020 Aug;26(8):1309. doi: 10.1038/s41591-020-0978-1. Epub 2017 May 24. Detailed Description: This is an international, single-arm, multicenter phase 2 trial, in Tafasitamab plus lenalidomide in relapsed or refractory diffuse large B-cell lymphoma (L-MIND): a multicentre, prospective, single-arm, phase 2 study. Thank you for submitting a comment on this article. official website and that any information you provide is encrypted Phase II trial of triple tyrosine kinase receptor inhibitor nintedanib in recurrent high-grade gliomas. 2020;323(9):84453. 2019;10(14):321423. BMC Medical Research Methodology Phase 2 study of Pomalidomide (CC-4047) monotherapy for children and young adults with recurrent or progressive primary brain tumors. mPFS does not include the events of disease progression but include the events of death within 3months after randomization. Phase 2 trial of temozolomide and pegylated liposomal doxorubicin in the treatment of patients with glioblastoma multiforme following concurrent radiotherapy and chemotherapy. 2021 Jan;20(1):117-128. doi: 10.1002/pst.2060. CAS Bethesda, MD 20894, Web Policies Tawbi HA, et al. 1989;10(1):110. Loncastuximab tesirine in relapsed or refractory diffuse large B-cell lymphoma (LOTIS-2): a multicentre, open-label, single-arm, phase 2 trial Summary Careers. None Created on Jan 16, 2023 Publication details Reviews All publications My publications Add new publication Import from PubMed More WebDive into the research topics of 'MATRix-RICE therapy and autologous haematopoietic stem-cell transplantation in diffuse large B-cell lymphoma with secondary CNS involvement (MARIETTA): an international, single-arm, phase 2 trial'. Simon R. Optimal two-stage designs for phase II clinical trials. WebARTICLE Clinical Trials 2011; 8: 260269 Quantitative evaluation of single-arm versus randomized phase II cancer clinical trials Gregory R Ponda,b and Saqib Abbasia Background There is a debate among cancer researchers about the use of single-arm or randomized phase II clinical trial designs; however, there is limited published The https:// ensures that you are connecting to the Keywords: For good study design with phase II trials, investigators should maintain low level of type I and II errors. This systematic review has some limitations. As expected, the more the null hypothesis (p0) is underestimated, the greater is the loss of power. Finally, we can calculate the expected sample sizes of \({n}_{1}\) or \(n\) with a true response rate of the experimental therapy since the sample size of \({n}_{1}\) and \(n\) are random variables [6, 12, 17]. Please remove one or more studies before adding more. Why Should I Register and Submit Results? Webmultiple myeloma: a multicenter, single-arm phase 2 trial Thilo Zander 1 , Thomas Pabst 2 , Smi Schr 3 , Stefan Aebi 1,2 , Ulrich Mey 4 , Urban Novak 2 , Erika Lerch 5 , Galle Rhyner Agocs 6 , Even though Simons two-stage designs have been popularly in oncology Phase 2 trials during last two decades, none, to our knowledge, has examined the usage for published articles that had applied the two-stage designs in Phase 2 single-arm trials in brain tumors. This implies that most brain tumor clinical trials are at high risk with great uncertainty in trial outcomes. Neyns B, et al. Phase II trial of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma. sharing sensitive information, make sure youre on a federal Loncastuximab tesirine: an effective therapy for relapsed or refractory diffuse large B-cell lymphoma. 75 patients were Fortunately, several reviews present that PFS6 and OS are generally strongly associated in glioblastoma trials, but not between RR and OS [54]. Phase II and biomarker study of programmed cell death protein 1 inhibitor nivolumab and metronomic cyclophosphamide in paediatric relapsed/refractory solid tumours: Arm G of AcSe-ESMART, a trial of the European innovative therapies for children with cancer consortium. Phase 2 trial of hypoxia activated evofosfamide (TH302) for treatment of recurrent bevacizumab-refractory glioblastoma. J Immunother Cancer. Santoni M, et al. Careers. 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. Clinical end points in recurrent glioblastoma: are antiangiogenic agents friend or foe? 2019;85(6):E96774. In Wang Z-X, et al. IEAC-assessed median DOR was not reached (NR; 95% CI, 1.87 months to NR). To illustrate the effect of underestimated historical control on the study power, we carried out a simulation study as follows. Yoo, W., Kim, S., Garcia, M. et al. Glioblastoma: emerging treatments and novel trial designs. official website and that any information you provide is encrypted 2014;33(12):2004-2016. PRISMA 2020 explanation and elaboration: updated guidance and exemplars for reporting systematic reviews. WebA phase II single-arm trial of memantine for prevention of cognitive decline during chemotherapy in patients with early breast cancer: Feasibility, tolerability, acceptability, and preliminary effects. This phase 2 study assessed the efficacy and safety of the dual EZH1 and EZH2 inhibitor valemetostat in patients with R/R ATL. Epub 2022 Apr 1. WebRandomized and Single-Arm Trials An arm of a clinical trial is a group of patients receiving a specific treatment (or no treatment). Most frequently used population was adult patients with recurrent glioblastoma. There was no language restriction, but only complete papers published in peer-reviewed journals were considered. We identified a total of 81 articles based on online search using Two-stage Phase II trials in glioblastoma. Clipboard, Search History, and several other advanced features are temporarily unavailable. Altwairgi AK, et al. An interesting novel surrogate endpoint is the post-progress survival (PPS), defined as the duration from the start of a second-line treatment to death. Among examined 29 trials, 12 trials (41%) appropriately provided key input parameters and sample size results from two-stage design implementation. Which one should be chosen for our studies of the two? By doing so, the chance to capture the effectiveness of a treatment can be increased. Epub 2022 Dec 6. 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. In safety studies, a main concern is that there may be too many patients who experience an adverse event. BSK reports grants from ADC Therapeutics, during the conduct of the study; and personal fees from Seattle Genetics and Genentech, outside of the submitted work. 2008;14(12):367582. Cancers (Basel). Among 29 trials, only three has been completed for both stages and two studies have shown the efficacy. 2022 May;169:107420. doi: 10.1016/j.csda.2021.107420. Phase 0 clinical trials in oncology new drug development. Stat Med. Cancer immunotherapy trial design with random delayed treatment effect and cure rate. Google Scholar. Phase 2 study of bosutinib, a Src inhibitor, in adults with recurrent glioblastoma. PMC Accessibility 2009;27(19):30736. sharing sensitive information, make sure youre on a federal Studies a U.S. FDA-regulated Drug Product: Studies a U.S. FDA-regulated Device Product: Irinotecan, solution for infusion (20 mg/mL), Temozolomide, capsules (5 mg, 20 mg and 100 mg), The humanized immunoglobulin isotype G (IgG1) monoclonal antibody (mAb) naxitamab, solution for infusion (4 mg/mL), Sargramostim (GM-CSF), lyophilized 250 g single use vial (250 g/vial), Overall response rate (ORR) [TimeFrame:84 days], ORR after 2 cycles [TimeFrame:42 days], Duration of response (DoR) [TimeFrame:2 years], Complete response (CR) rate [TimeFrame:84 days], Time to first subsequent therapy [TimeFrame:3 years], Progression free survival (PFS) [TimeFrame:3 years], Overall survival (OS) [TimeFrame:3 years], Overall survival (OS) [TimeFrame:1 year], Overall survival (OS) at 2 years [TimeFrame:2 year], Receipt of Standard of Care (SoC) frontline induction/consolidation therapy (including surgery, chemotherapy, ASCT, MIBG, radiotherapy, immunotherapy, or retinoids), verified first progression during multi-drug frontline treatment or, verified first episode of relapse, defined as recurrence after response to frontline treatment, or, verified first designation of refractory disease, defined as persistent metastatic disease (SD or minor response by INRC and MIBG curie score 3) detected at conclusion of at least 4 cycles of multi-drug induction chemotherapy on or according to a high-risk NB treatment protocol as defined above, Measurable tumor on CT/MRI scan that is MIBG-avid or demonstrates increased FDG uptake on PET scan, MIBG (Metaiodobenzylguanidine) scan with positive uptake at a minimum of one site. doi: 10.1016/S2352-3026(21)00311-2. 2015;34(16):240316. If advanced to stage 2, the number of patients in both stages (\(n\)) and the response number (\(r\)) of the efficacy for the Phase 2 trial are provided. Listing a study does not mean it has been evaluated by the U.S. Federal Government. By using this website, you agree to our Intrathecal administration of trastuzumab for the treatment of meningeal carcinomatosis in HER2-positive metastatic breast cancer: a systematic review and pooled analysis. In this systematic review, we have examined 29 studies from Phase 2 single-arm two-stage trials in glioblastoma to assess the appropriateness and transparency of the study design and sample size calculation of Phase 2 single-arm two-stage trials in glioblastoma. Disclaimer, National Library of Medicine Biometrics. 2022. Du XJ, et al. Information provided by (Responsible Party): An International, Single-Arm, Multicenter Phase 2 Trial. 2003;95(19):1434-1439. Careers. 2022 Dec 7;14(24):6026. doi: 10.3390/cancers14246026.
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