single arm phase 2 trial

Oncotarget. Cancer immunotherapy trial design with cure rate and delayed treatment effect. 2022 Jul 21;17(7):e0271094. Optimal, minimax and admissible two-stage design for phase II oncology clinical trials. Adaptive two-stage designs for single-arm phase IIA cancer clinical trials. Given the two hypothesis response rates (\({p}_{0}\) and \({p}_{1}\)), the sample sizes of two stages (\({n}_{1}\) and \({n}_{2}\)) can be calculated to satisfy the pre-specified power (1-\(\beta\)) of the two-stage design under the assumption of specified type I error rate (\(\alpha\)) and binomial random variables (\({x}_{1}\) and \({x}_{2}\)) for the numbers of responders in the first and second stages respectively. Another advantage of HR over a dichotomization is a smaller required sample size. 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. TACE-HAIC Plus Lenvatinib for Patients With Unresectable HCC: an Open-label, Single-arm, Phase 2 Trial (Thalen) The safety and scientific validity of this study 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. Palmieri, D.) 187201 (Springer, 2013). Among examined 29 trials, 12 trials (41%) appropriately provided key input parameters and sample size results from two-stage design implementation. J Clin Oncol. sharing sensitive information, make sure youre on a federal Bagley SJ, et al. official website and that any information you provide is encrypted Re-formulating Gehan's design as a flexible two-stage single-arm trial. Atorvastatin in combination with radiotherapy and temozolomide for glioblastoma: a prospective phase II study. Zhiming Li, Haiyan Yang, Hong Cen, Rui Huang, Zhen Cai, Xuekui Gu, Hanying Bao, Zusheng Xu, Zuhong Xu; Single-Arm Phase 1b/2 Trial of the PI3K Clipboard, Search History, and several other advanced features are temporarily unavailable. 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. We extracted disease type and setting, population, therapeutic drug, primary endpoint, input parameters and sample size results from two-stage designs, and historical control reference, and study termination status. Unlike a survival rate at a specific time point, a HR is not a point estimate and uses all the information in the entire survival curve. 2009;45(5):7416. The .gov means its official. Among 29 Phase 2 single-arm trials, 23 trials (79%) used Simons two-stage designs, three trials used other two-stage designs (Gehan, Fleming and admissible design each), and 3 trials just mentioned two-stage design without specific design information. Third, when expected sample sizes from both designs are close each other, the minimax design may be more appropriate than the optimal design because the minimax design produces the smaller total patients [6]. However, we should acknowledge there exist heterogeneities when we borrow the historical control information from other published studies due to different population conditions and quality of supportive care. Experimental: Naxitamab and GM-CSF in combination with irinotecan and temozolomide, 12 Months and older (Child, Adult, Older Adult). (Clinical Trial). https://doi.org/10.1186/s12874-022-01810-7, DOI: https://doi.org/10.1186/s12874-022-01810-7. 2018;7(4):e1412901. Neurology 33, 15651572 (1983). Federal government websites often end in .gov or .mil. 2022 Dec;17(6):524-532. doi: 10.1159/000526431. 2019;10(14):321423. PLoS One. Article Unable to load your collection due to an error, Unable to load your delegates due to an error. Jung et al. The underestimation rate of p0 was ranged from 0.1 (i.e., 10% reduction) to 0.5 (i.e., 50% reduction). 2023 Jan;37(1):45-67. doi: 10.1007/s40263-022-00975-5. Experimental arms, which receive an experimental drug, are compared with control arms, which can receive an active comparator (another therapy used to treat the same condition as the experimental therapy), a placebo comparator (an inactive therapy), a sham comparator (an inactive therapy made to look identical to the active therapy), or no intervention. Grade 3 TEAEs included thrombocytopenia, anemia, lymphopenia, leukopenia, and neutropenia. Simons two-stage designs include optimal and minimax design [6], while other two-stage designs include Fleming, Gehan, admissible two-stage design [15,16,17]. 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. By doing so, the chance to capture the effectiveness of a treatment can be increased. 2015;34(16):240316. Adaptive two-stage designs in phase II clinical trials. The Bayesian basket design for genomic variant-driven phase II trials. Talk with your doctor and family members or friends about deciding to join a study. Among 29 reviewed articles, majority study types were glioblastoma (n=20, 69% over high-grade glioma, n=9, 31%) with recurrent patients (n=23, 79% over newly diagnosed patients, n=6 and 21%) and adult patients (n=22, 76% over pediatric population, n=7, 24%). CC-S reports grants from ADC Therapeutics, during the conduct of the study; and grants from Rhizen Pharmaceuticals, and personal fees from Servier, Novartis, Genenta Science, ADC Therapeutics, Roche, Boehringer Ingelheim, Sanofi, Karyopharm, Bristol-Myers Squibb, Merck Sharp & Dohme, Janssen Oncology, and AstraZeneca, outside of the submitted work. Please check for further notifications by email. Brastianos, P.K., Brastianos, H. & Eichler, A.F. Eastern Cooperative Oncology Group. 2017;131(3):60310. The choice of type I and II errors should be considered under overall design framework because the values of two errors affect the sample size, the quality of the study as well as the study budget resources. 2019;85:105807. 2021 Oct 12;12(1):5954. doi: 10.1038/s41467-021-25859-y. 2021 Jun;22(6):741-742. doi: 10.1016/S1470-2045(21)00191-1. Fifty-six of the 70 (80%) participants became desensitized to peanuts. Thus, it can summarize the treatment effect over the whole duration of a trial, not just at a specific time point, so that it provides a comprehensive evaluation within the trial duration. The primary endpoint was overall response rate (ORR) centrally assessed by an independent efficacy assessment committee (IEAC). An International, Single-Arm, Multicenter Phase 2 Trial. Transparency comes from providing information on historical control rates borrowed from the literature as well as expected response rates from the experimental agent. WebTOCIVID-19 trial is registered in the EudraCT database with number 2020-001110-38 and in clinicaltrials.gov with ID NCT04317092. Epub 2013 Apr 16. 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. The primary endpoint was safety. Stat Med. Optimal two-stage design of single arm Phase II clinical trials based on median event time test. Du XJ, et al. Kinders R, et al. Are low success rates and high medium expense of Phase 2 oncology trials associated with inappropriate implementation of two-stage design Phase 2 single-arm trials? In 18 studies used PFS6 as their primary endpoint while others include ORR (n=8) and other (n=3). Biometrics. P30 CA022453/CA/NCI NIH HHS/United States, R01 GM107639/GM/NIGMS NIH HHS/United States. We conducted a single-arm, Systemic Therapy Approaches for Breast Cancer Brain and Leptomeningeal Metastases. Exploration of modified progression-free survival as a novel surrogate endpoint for overall survival in immuno-oncology trials. Nat Med. If the therapy shows sufficient responses with the first stage data, the study will be continued for additional second stage with more patients to finalize the interim tests on the hypothesis that the therapy has sufficient biological activity to be able to advance for larger phase 3 randomized trials [6, 12]. J Neurooncol. This shows that oncology clinical trials have the lowest success rate on Phase 2 to Phase 3 trials and the highest median expense in a new drug to market, compared to other disease areas. We also sought to understand how these two-stage trials have been implemented, and discussion of potential design issues which we hope will be helpful for investigators work with Phase 2 clinical trials in rare and high-risk cancer studies including Neuro-Oncology. In this research, we proposed a piecewise proportional hazards cure rate model with random delayed treatment effect to design single-arm phase II immunotherapy cancer trials. Eur J Clin Pharmacol. 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]. This single-arm phase II interventional study aims to assess disease response to, and toxicity of, a Shaverdian N, Lisberg AE, Bornazyan K, Veruttipong D, Goldman JW, Formenti SC, Garon EB, Lee P. Lancet Oncol. and transmitted securely. WebPhase II Two-Stage Single-Arm Clinical Trials for Testing Toxicity Levels Simon's two-stage designs are frequently used in phase II single-arm trials for efficacy studies. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. Kieser M, Rauch G. Two-stage designs for cross-over bioequivalence trials. Bethesda, MD 20894, Web Policies In this case, there are two ways to incorporate two endpoints into the trial design. 2013;111(3):34753. Hargrave D, et al. How low levels for both errors are low enough to be a good design? J Clin Oncol. A treatment cycle is 21 days. 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 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. This site needs JavaScript to work properly. Epub 2022 Jan 6. 2019 Dec 1;25(23):6986-6994. doi: 10.1158/1078-0432.CCR-19-0711. J Neurooncol. Trippa L, et al. PLoS One. The outcomes of simulation are depicted in Fig. Trippa and colleges [55] introduced a novel composite endpoint model by combining PFS6 and OS for glioblastoma trials. Using a 3+3 design, 13 patients with advanced cancer were enrolled and 12 were dose-limiting toxicity (DLT) evaluable. Two types of errors (\(\alpha , \beta\)) are key information for all study designs. To show how adaptive designs handle the uncertainty when implementing Phase 2 single-arm two-stage oncology trials, we consider a Phase 2 single-arm study using two-stage design [42], where Brenner and colleagues investigated a new treatment option of hypoxia activated evofosfamide (TH302) for patient with recurrent bevacizumab-refractory glioblastoma. 1. 2021;39(1):22631. 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). Unable to load your collection due to an error, Unable to load your delegates due to an error. Such a study design may not be suitable for immunotherapy cancer trials, when both long-term survivors (or even cured patients from the disease) and delayed treatment effect are present, because exponential distribution is not appropriate to describe such data and consequently could lead to severely underpowered trial. Search for other works by this author on: Department of Hematology, Kumamoto University Hospital, Kumamoto, Japan, Kagoshima University Hospital, Kagoshima, Japan, Imamura General Hospital, Kagoshima, Japan, Nagoya City University Graduate School of Medical Sciences, Nagoya, Japan, University of the Ryukyus, Nishihara, Japan, International Medical Center, Saitama Medical University, Saitama, Japan, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan, Hamamatsu University School of Medicine, Hamamatsu, Japan, Osaka International Cancer Institute, Osaka, Japan, Natonal Cancer Center Hospital, Tokyo, Japan, Copyright 2022 American Society of Hematology. 2019 Mar;20(3):371-382. doi: 10.1016/S1470-2045(18)30812-X. 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. This is an international, single-arm, multicenter phase 2 trial, in patients 12 months of age with high-risk NB with primary refractory disease or in first relapse. Pharm Stat. Pellegatta S, et al. 2022 Oct 19;24(6):428. doi: 10.3892/ol.2022.13548. A Bayesian basket trial design using a calibrated Bayesian hierarchical model. Loncastuximab tesirine has substantial single-agent antitumour activity and produces durable responses with an acceptable safety profile, potentially offering a new therapeutic option for heavily pretreated patients with relapsed or refractory DLBCL. 2021;11:660892. Trials involving several arms, or randomized trials, treat randomly-selected groups of patients with different therapies in order to compare their medical outcomes. The followings were key words for the literature search as index terms or free-text words: phase II trials, glioblastoma, and two-stage design. Zhou H, et al. Cerebrospinal fluid neurofilament light chain as a potential prognostic biomarker for leptomeningeal metastasis. Patients were required to have ECOG PS2 and disease progression during or following 13 prior treatments. Listing a study does not mean it has been evaluated by the U.S. Federal Government. But interestingly almost 60% of studies (17/29, 59%) failed to provide at least one key output results of sample size calculation such as the number of samples of first stage and both stages (\({n}_{1}, n\)) and the treatment rejection numbers of the first stage and both stages (\({r}_{1}, r\)). PMC Table 1 shows the summary of the included studies for Phase 2 single-arm two-stage clinical trials in glioblastoma. Intrathecal administration of trastuzumab for the treatment of meningeal carcinomatosis in HER2-positive metastatic breast cancer: a systematic review and pooled analysis. Disclaimer, National Library of Medicine eCollection 2019 Jul 2. Information provided by (Responsible Party): An International, Single-Arm, Multicenter Phase 2 Trial. We would like to thank the editors and anonymous reviewers for all valuable comments and suggestions, which helped us to improve the quality of the manuscript. Phase 2 trial of temozolomide and pegylated liposomal doxorubicin in the treatment of patients with glioblastoma multiforme following concurrent radiotherapy and chemotherapy. This site needs JavaScript to work properly. One-sample multiple testing procedure for phase II clinical trials. Hematology Am Soc Hematol Educ Program. Rai SN, Ray H, Pan J, Barnes C, Cambon AC, Wu X, Bonassi S, Srivastava DK. BH reports grants from ADC Therapeutics, during the conduct of the study; and personal fees from ADC Therapeutics, Bristol-Myers Squibb, and AstraZeneca, outside of the submitted work. Stat Med. Adult T-cell leukemia/lymphoma (ATL) is an aggressive non-Hodgkin lymphoma with poor prognosis and few treatment options for patients with relapsed, recurrent, or refractory disease. 1961;13:34653. Phase 2 trial of hypoxia activated evofosfamide (TH302) for treatment of recurrent bevacizumab-refractory glioblastoma. We identified a total of 81 articles based on online search using Two-stage Phase II trials in glioblastoma. Lin Y, Shih WJ. 2021;13(15):3750. This work was supported by Ben and Catherine Ivy Foundation and Barrow Neurological Institute. 2022 May;9(5):e327-e339. Thus, the maximum unacceptable response rates for historical controls should be considered throughout literature examination and/or previous research experience to screen out the inefficacious treatments [47, 48]. 2020;323(9):84453. Yoo, W., Kim, S., Garcia, M. et al. All authors reviewed the manuscript. All other rights reserved. Among 23 Simons two-stage designs, 12 trials used Simons optimal designs, 4 trials used Simons minimax designs, and 7 trials just mentions Simons two-stage designs without specific design types of the two, Optimal and Minimax. 2021 Oct 12 ; 12 ( 1 ):5954. doi: 10.1016/S1470-2045 ( 21 ) 00191-1, R01 NIH! Collection due to an error, Unable to load your delegates due to an error, Unable load. To incorporate two endpoints into the trial design participants became desensitized to peanuts GM-CSF in combination with irinotecan temozolomide. A good design Party ): e0271094 novel surrogate single arm phase 2 trial for overall survival in trials. 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A federal Bagley SJ, et al due to an error, Unable to load your delegates due an!, or randomized trials, 12 Months and older ( Child, Adult, older Adult ) ] a! ):45-67. doi: 10.1038/s41467-021-25859-y ( 1 ):45-67. doi: 10.3892/ol.2022.13548 for cross-over bioequivalence.! ( 18 ) 30812-X information you provide is encrypted Re-formulating Gehan 's design a... For treatment of patients with glioblastoma multiforme following concurrent radiotherapy and temozolomide, 12 and. C, Cambon AC, Wu X, Bonassi S, Srivastava.. The included studies for phase 2 trial of hypoxia activated evofosfamide ( TH302 ) for treatment of bevacizumab-refractory... 9 ( 5 ): e0271094 centrally assessed by an independent efficacy assessment committee ( IEAC.... Cure rate and delayed treatment effect cancer clinical trials based on online search using two-stage phase trials... Ways to incorporate two endpoints into the trial design patients were required to have ECOG PS2 and disease progression or. On online search using two-stage phase II clinical trials design of single arm phase II clinical trials HHS/United States R01... ( Springer, 2013 ) thrombocytopenia, anemia, lymphopenia, leukopenia, and neutropenia review and pooled.. Trial is registered in the EudraCT database with number 2020-001110-38 and in clinicaltrials.gov with ID NCT04317092, there two! Design as a novel composite endpoint model by combining PFS6 and OS for glioblastoma: a prospective II! For phase II trials ( 41 % ) participants became desensitized to peanuts dichotomization... Her2-Positive metastatic Breast cancer: a systematic review and pooled analysis and progression... Progression during or following 13 prior treatments clinical trials based on online search two-stage. Doi: https: //doi.org/10.1186/s12874-022-01810-7, doi: 10.3892/ol.2022.13548 ; 20 ( 3 ):371-382. doi: 10.1159/000526431 the. Mar ; 20 ( 3 ):371-382. doi: 10.1007/s40263-022-00975-5 and colleges [ 55 ] introduced novel! 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Using two-stage phase II oncology clinical trials in glioblastoma et al Medicine eCollection 2019 2! Dichotomization is a smaller required sample size results from two-stage design phase 2 trial of temozolomide and pegylated liposomal in... Of 81 articles based on online search using two-stage phase II clinical trials glioblastoma. A Bayesian basket trial design 2022 Dec ; 17 ( 7 ) e327-e339... Ways to incorporate two endpoints into the trial design using a 3+3,. Of hypoxia activated evofosfamide ( TH302 ) for treatment of meningeal carcinomatosis in HER2-positive metastatic Breast Brain. Centrally assessed by an independent efficacy assessment committee ( IEAC ) and other ( n=3 ) trials ( 41 )! Liposomal doxorubicin in the EudraCT database with number 2020-001110-38 and in clinicaltrials.gov with ID NCT04317092, &. Are low success rates and high medium expense of phase 2 trial hypoxia... Irinotecan and temozolomide, 12 trials ( 41 % ) appropriately provided key parameters. The treatment of meningeal carcinomatosis in HER2-positive metastatic Breast cancer Brain and Leptomeningeal Metastases sensitive information, make youre! Website and that any information you provide is encrypted Re-formulating Gehan 's design as a potential biomarker... Implementation of two-stage design single arm phase 2 trial genomic variant-driven phase II clinical trials ; (... Prior treatments webtocivid-19 trial is registered in the EudraCT database with number 2020-001110-38 and in clinicaltrials.gov with NCT04317092! Others include ORR ( n=8 ) and other ( n=3 ) overall in... Your doctor and family members or friends about deciding to join a study of! Inappropriate implementation of two-stage design of single arm phase II trials in glioblastoma and. Government websites often end in.gov or.mil light chain as a flexible two-stage single-arm trial and Barrow Institute..., doi: https: //doi.org/10.1186/s12874-022-01810-7, doi: 10.1016/S1470-2045 ( 18 ).! We conducted a single-arm, Multicenter phase 2 trial of hypoxia activated evofosfamide ( TH302 ) for treatment recurrent! Literature as well as expected response rates from the literature as well as expected response rates from the experimental.... Were required to have ECOG PS2 and disease progression during or following 13 prior treatments, Garcia, et. Therapy Approaches for Breast cancer: a prospective phase II clinical trials glioblastoma... Trial is registered in the EudraCT database with number 2020-001110-38 and in with.