single arm phase 2 trial

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 2015 May-Jun;14(3):226-32. doi: 10.1002/pst.1678. Epub 2017 Aug 16. Epub 2022 Jan 21. Single-arm or non-randomized trials,in which everyone enrolled in a trial receives the experimental therapy, are common in Phase 1 and 2 testing. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. doi: 10.1371/journal.pone.0246448. The .gov means its official. However, it is important that efficient and valid study designs be applied successfully and adequately to reach the aims of phase 2 oncology trials. Inclusion of non-inferiority analysis in superiority-based clinical trials with single-arm, two-stage Simon's design. A recent systematic review shows that both RR and PFS6 are suitable surrogate endpoints for OS, but their surrogacy varies according to therapy line or type and study size [53]. Phase 2 study of Pomalidomide (CC-4047) monotherapy for children and young adults with recurrent or progressive primary brain tumors. Appropriate selection on primary endpoint, transparency of historical control and experimental rates, right implementation for two-stage design and sample size calculation, potential incorporation of adaptive designs, and utilization of Phase 0 paradigm [65, 67,68,69,70] might help overcoming the challenges on glioblastoma therapeutic trials in Phase 2 trials. When the sample size is limited, one can use the Bayesian optimal design for phase II clinical trials, in which the power will be maximized given the fixed sample size and choice of type I error rate [51]. 1990;9(3):21528. 2019;85:105807. In this study, we systematically reviewed published single-arm two-stage Phase 2 trials for patients with glioblastoma and high-grade gliomas (including newly diagnosed or recurrent). Suppose both RR and PFS6 are considered primary endpoints. Trippa and colleges [55] introduced a novel composite endpoint model by combining PFS6 and OS for glioblastoma trials. (Clinical Trial). 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. The median follow-up of surviving patients was 6.3 months (range, 2.2-12.5 months). Phase II open-label study of nintedanib in patients with recurrent glioblastoma multiforme. If it may be available to enroll only ten patients per year, the optimal design needs a half year longer than the minimax design. 2019;8(13):598894. When there is uncertainty among endpoints as a surrogate for OS, several primary endpoints can be employed together. Multicenter Phase II Study of Lenalidomide in Relapsed or Recurrent Adult T-Cell Leukemia/Lymphoma: ATLL-002. Brenner AJ, et al. 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 (\({r}_{1}, r\)). 2019;144(2):35968. WebA Single-arm, Multicenter, Phase 2 Trial to Evaluate Efficacy and Safety of Lenvatinib in Combination With Everolimus in Subjects With Unresectable Advanced or Metastatic Systemic Therapy Approaches for Breast Cancer Brain and Leptomeningeal Metastases. Among 29 trials, only three has been completed for both stages and two studies have shown the efficacy. Kalpathy-Cramer J, et al. Stat Med. Epub 2022 Jan 6. Stat Med. J Clin Neurosci. Cancer immunotherapy trial design with long-term survivors. 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. J Clin Oncol. Reference curve sampling variability in one-sample log-rank tests. Cite this article. Unable to load your collection due to an error, Unable to load your delegates due to an error. Privacy 2022 Feb 20;41(4):786-797. doi: 10.1002/sim.9258. Comput Stat Data Anal. Lastly, this study focuses on right implementation for the study design and sample size calculation. Cerebrospinal fluid neurofilament light chain as a potential prognostic biomarker for leptomeningeal metastasis. Phase II trial of triple tyrosine kinase receptor inhibitor nintedanib in recurrent high-grade gliomas. -, Grossman, S. A. et al. 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]. Loncastuximab tesirine in relapsed or refractory diffuse large B-cell lymphoma (LOTIS-2): a multicentre, open-label, single-arm, phase 2 trial Summary 2019;20(2):27386. Accessibility Comparing survival of a sample to that of a standard population. Thall PF, Simon R. Incorporating historical control data in planning phase II clinical trials. Particularly, a recent research of the phase 2 basket trials has extended the two-stage design for multiple heterogeneous indications, which is an important tool to identify the effective drug through a generalized framework of an optimal basket design. Transparency comes from providing information on historical control rates borrowed from the literature as well as expected response rates from the experimental agent. government site. Page MJ, et al. eCollection 2022 Dec. Transl Cancer Res. EG-Mirotin safety and efficacy were investigated in patients with type 1 or type 2 diabetes mellitus and moderate to severe NPDR. The site is secure. Copyright 2022 American Society of Hematology. Analysis of survival data under the proportional hazards model. Eur J Cancer. The most widely used endpoints were PFS6 (n=18) and ORR (n=8) in Phase 2 single-arm trials. We evaluated the efficacy and safety of valemetostat, a potent EZH1 and EZH2 inhibitor, in treating relapsed/refractory (R/R) ATL. Better understanding on the study drug information and targeted molecular information might help the understanding of why the two-stage trials had been terminated after the stage 1 and ineffective after successful completion of both stages. The .gov means its official. The https:// ensures that you are connecting to the A recent paper used two drugs of Nivolumab (NIV) and Cyclophosphamide (CYC) for the combination therapeutic treatment [44]. Before Using a 3+3 design, 13 patients with advanced cancer were enrolled and 12 were dose-limiting toxicity (DLT) evaluable. WebZhao, Y., Zhang, X., Jin, C.X., et al. Optimal two-stage designs for exploratory basket trials. 2019;144(2):4037. Alternatively, novel surrogates can be used instead of RR and PFS6. Epub 2021 May 11. Article 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 Novak2, Erika Lerch 5, Galle Rhyner Agocs 6, 2022;169:107420. Spatial two-stage designs for phase ii clinical trials. Cancer immunotherapy trial design with cure rate and delayed treatment effect. Tawbi HA, et al. Drugs. Neyns B, et al. 2021;13(15):3750. J Chronic Dis. Thus, PFS6 should be considered the primary endpoint over RR whenever possible. 2015;34(6):1031-1040. The primary endpoint was safety. 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 , Most studies (n=27, 93%) provided the number of patients in stage 1 and both stages, so many trials (n=17) failed to report one or more from both response numbers of stage 1 and both stages which are key information to determine the study continuation toward the second stage (\({r}_{1}\)) at the end of first stage and hypothesis testing of efficacy (\(r\)) at the end of second stage. Simon R. Optimal two-stage designs for phase II clinical trials. An increasing fraction of patients with metastatic cancer develop leptomeningeal dissemination of disease (LMD), and survival is dismal1-3. OPTIM-ARTSAn adaptive phase II open platform trial design with application to a metastatic melanoma 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 2021;13(3):28694. Study design input information and output results from sample size calculation related to two-stage design implementation were examined. 1961;13:34653. Intraventricular methotrexate therapy of leptomeningeal metastasis from breast carcinoma. Pembrolizumab plus trastuzumab in trastuzumab-resistant, advanced, HER2-positive breast cancer (PANACEA): a single-arm, multicentre, phase 1b-2 trial. Among examined 29 trials, 12 trials (41%) appropriately provided key input parameters and sample size results from two-stage design implementation. We also sought to understand how these two-stage trials have been implemented and discussed potential design issues which we hope will be helpful for investigators who work with Phase 2 clinical trials in rare and high-risk cancer studies including Neuro-Oncology. An armof a clinical trial is a group of patients receiving a specific treatment (or no treatment). We examined following information: (1) general study design information (study name, first author, publication year, disease type and status, patient type, therapeutic drug type, and primary endpoint type), (2) the design type and key input information for the implementation of two-stage designs (design type, type I and II error rates, unacceptable and acceptable response rates), (3) key results from the sample size calculation for two-stage design (the number of patients for stage one and both stages, and the rejection numbers for hypothesis tests at the ends of stage one and second stage), and (4) reference of historical control rates applied. 2021 Dec;8(12):e891-e901. FOIA Stat Biopharma Res. 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. 2015;34(16):240316. Keywords: A Phase I Study of ADCT-402 (Loncastuximab Tesirine), a Novel Pyrrolobenzodiazepine-Based Antibody-Drug Conjugate, in Relapsed/Refractory B-Cell Non-Hodgkin Lymphoma. Multistage designs for phase II clinical trials: statistical issues in cancer research. The primary endpoint was safety. Le Rhun, E. et al. The followings were key words for the literature search as index terms or free-text words: phase II trials, glioblastoma, and two-stage design. Sci Rep. 2021;11(1):2306. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. Would you like email updates of new search results? Accessibility Based on this, we tested the activity and safety of alternate-day dosing of 4 mg pomalidomide on a 28/28 day schedule in a multicenter, open-label phase 2 trial Cancer 124, 2135 (2018). Bendamustine treatment of Chinese patients with relapsed indolent non-Hodgkin lymphoma: a multicenter, open-label, single-arm, phase 3 study. 2017 Jul;18(7):895-903. doi: 10.1016/S1470-2045(17)30380-7. Copyright 2021 Elsevier Ltd. All rights reserved. government site. Would you like email updates of new search results? Table 1 is the summary of the included studies [18,19,20,21,22,23,24,25,26,27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46]. Phase II trial of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma. By doing so, the chance to capture the effectiveness of a treatment can be increased. Nat Commun. Epub 2022 Dec 6. Valemetostat demonstrated promising efficacy and tolerability in heavily pretreated patients, warranting further investigation in treating R/R ATL. Spatial Two-stage Designs for Phase II Clinical Trials. Two types of errors (\(\alpha , \beta\)) are key information for all study designs. Neuro Oncol. Federal government websites often end in .gov or .mil. Lassman AB, et al. Invest New Drugs. Kummar S, et al. 2018;74(2):529-537. Second, given the limited number of available single-arm two-stage trials, the results in these studies are subject to specific degree of selection criteria. The sample size calculation for the two-stage designs is determined based on the historical control rate and difference between the two rates (\({p}_{0}, {p}_{1}\)). The underestimation rate of p0 was ranged from 0.1 (i.e., 10% reduction) to 0.5 (i.e., 50% reduction). Thus, the key input parameters (\(\alpha , \beta , {p}_{0}, {p}_{1}\)) and output results (\({n}_{1}, n, {r}_{1}, r\)) were investigated to assess the appropriate usage and report of the two-stage phase 2 trials. A Phase 2 single-arm study using two-stage design [42], where a new treatment option of hypoxia activated evofosfamide (TH302) for patient with recurrent bevacizumab-refractory glioblastoma was investigated. 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. KH, JF, SH, and YQ report personal fees from ADC Therapeutics, during the conduct of the study. The percentage of patients who experienced one (or more) grade 3 or higher adverse events at least possibly related to treatment was 40%, the most frequent being hyperglycemia (n = 6), nausea (n = 7) and vomiting (n = 7). CAS Muhic A, et al. Only three trials (11%) provided key input parameters, appropriately reported output results from sample size calculation of two-stage designs, and finally provided the reference and explanation of historical control rates. Sampayo-Cordero M, Miguel-Huguet B, Prez-Garca J, Pez D, Guerrero-Zotano L, Garde-Noguera J, Aguirre E, Holgado E, Lpez-Miranda E, Huang X, Malfettone A, Llombart-Cussac A, Corts J. Contemp Clin Trials Commun. Randomized prospective comparison of intraventricular methotrexate and thiotepa in patients with previously untreated neoplastic meningitis. Twenty-five patients (median age, 69.0) with a median of 3 prior lines of therapy were enrolled; 24 had prior mogamulizumab treatment. Article P30 CA022453/CA/NCI NIH HHS/United States, R01 GM107639/GM/NIGMS NIH HHS/United States. Disease population was categorized into three diseases of glioblastoma (n=20), high-grade glioma (n=8), and brain metastasis from glioblastoma (n=1), two settings of recurrent status (n=23) and newly diagnosed status (n=6), two patient types of adults (n=23) and child or pediatric (n=6), and two therapeutic drug types of single (n=17) and combination (n=12). Bagley SJ, et al. The other three trials used two-stage designs like Inadmissible design, Fleming and Gehan designs. Pembrolizumab in Patients With Extensive-Stage Small-Cell Lung Cancer: Results From the Phase Ib KEYNOTE-028 Study. Glioblastoma: emerging treatments and novel trial designs. 2020 Mar 15;39(6):698-708. doi: 10.1002/sim.8440. An International, Single-Arm, Multicenter Phase 2 Trial. XZ reports other from ADC Therapeutics, outside of the submitted work. (Yes, No), (8) all four output results of sample size appropriately reported? The primary endpoint was overall response rate (ORR) centrally assessed by an independent efficacy assessment committee (IEAC). 7 Briefly, SAUL ( NCT02928406) was a single-arm phase b study of atezolizumab in a real-world patient population with locally advanced or metastatic UC or non-UC of the urinary tract. 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. Please enable it to take advantage of the complete set of features! The statistical power conditional on the interim analysis of second stage is 97.3% for the true rate of 28.9%, which is far beyond the originally pre-specified 80% of power. Phase II trial of an AKT inhibitor (perifosine) for recurrent glioblastoma. 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]. 2021 Jun;22(6):741-742. doi: 10.1016/S1470-2045(21)00191-1. 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. We conduct a multi-center single-arm phase 2 trial to test the efficacy and safety of an oral neoadjuvant therapy with pyrotinib, letrozole and dalpiciclib (a CDK4/6 2022 May;169:107420. doi: 10.1016/j.csda.2021.107420. Another option of two-stage designs is admissible design or spatial design, which came from an idea Can we find a good alternative design between the minimax and optimal design. J Neurooncol. Pembrolizumab is safe and feasible and displays promising activity in patients with LMD. 1R01CA227156-01/U.S. A common practice in sample size calculation is Dowlati A, Crosby L, Remick SC, Makkar V, Levitan N. Lung Cancer. and transmitted securely. Chamberlain MC, Johnston SK. 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? Geoerger B, et al. Anticancer Res. Brastianos, P.K., Brastianos, H. & Eichler, A.F. WebThis single-arm, phase I trial evaluated NHS-IL12 12 g/kg SC q2w or 16.8g/kg SC q2w in patients with metastatic solid tumors. Ott PA, Elez E, Hiret S, Kim DW, Morosky A, Saraf S, Piperdi B, Mehnert JM. J. Neurooncol. Rai SN, Ray H, Pan J, Barnes C, Cambon AC, Wu X, Bonassi S, Srivastava DK. Hematology Am Soc Hematol Educ Program. 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. Blood 2022; blood.2022016862. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. 2022 Dec 26;56:101779. doi: 10.1016/j.eclinm.2022.101779. The patients will receive irinotecan 50 mg/m2/day IV and temozolomide 100 mg/m2/day orally (both on Days 1-5) in combination with naxitamab 2.25 mg/kg/day IV (Days 2, 4, 8 and 10) (total 9 mg/kg per cycle), and GM-CSF 250 ug/m2/day sc, (Days 6-10). The synonyms and closely related words include phase 2 for phase II trials, GBM or high-grade glioma for glioblastoma, and 2-stage, Simon, Fleming or Gehan for two-stage design. 2017;131(3):60310. J Neurooncol. Right implementation for two-stage design and sample size calculation, transparency of historical control and experimental rates, appropriate selection on primary endpoint, potential incorporation of adaptive designs, and utilization of Phase 0 paradigm might help overcoming the challenges on glioblastoma therapeutic trials in Phase 2 trials. Only around 41% of trials (n=12) appropriately provided the key input and output information for the study design and sample size calculation of two-stage designs phase 2 trials. Clipboard, Search History, and several other advanced features are temporarily unavailable. 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. Neuro Oncol. 2013;10(5):72034. Unable to load your collection due to an error, Unable to load your delegates due to an error. Re-formulating Gehan's design as a flexible two-stage single-arm trial. Temozolomide (TMZ) was mostly used for combination therapeutic drugs (n=7 with pegylated liposomal doxorubicin (PLD), O6-benzylguanine (O6B), irinotecan (IRI), decitabine (DAC), Dendritic (DEN), Nintedanib (NIN), and Atorvastatin (ATO)) while Bevacizumab (BEV) was second mostly used for combination drugs (n=3 with temsirolimus (TEM), Ponatinib (PON), and Evofosfamide (EVO)). (Yes, No), (10) all key input and output information as well as reference of historical control rates provided (Yes, No)?, and (11) did the trial be stopped (Yes, No)? Disclaimer, National Library of Medicine 2001 May;32(2):155-62. doi: 10.1016/s0169-5002(00)00220-8. 2013;113(1):12734. Copyright 2020 Friends of Cancer Research. eCollection 2019 Jul 2. Hargrave D, et al. The ability to stop a trial early is important to avoid therapeutic inefficiency for patients and to reduce the overall cost of clinical trials at this stage. 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. Br J Cancer. 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. 2005;23(28):7199206. Please enable it to take advantage of the complete set of features! 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. Bethesda, MD 20894, Web Policies Nonetheless, none has examined the usage for published articles that had applied the two-stage designs in Phase 2 single-arm trials in brain tumor. ADC Therapeutics. 2012;110(1):1118. Google Scholar. 11, 561569 (1993). TEAEs that occurred in 20% of patients included thrombocytopenia, anemia, alopecia, dysgeusia, neutropenia, lymphopenia, leukopenia, decreased appetite, and pyrexia. Adaptive two-stage designs for single-arm phase IIA cancer clinical trials. Chu Y, Yuan Y. Wong CH, Siah KW, Lo AW. Moreover, the success rates for phase 1 to approval, phase 2 to approval, and phase 3 to approval were 3.4%, 6.7% and 35.5%, respectively, in oncology therapeutic area, which were relatively low compared to other therapeutic areas (e.g., 25.5%, 32.3%, and 62.2% of cardiovascular disease therapeutic area and 25.2%, 35.1%, and 75.3% of infectious disease therapeutic area) [3]. Di Nunno V, et al. Bookshelf We here considered a single-arm single-stage design with a binary endpoint to achieve 80% power at a 1-sided Type 1 error rate of 5% (Fig. Neurology 33, 15651572 (1983). WebSingle-dose carboplatin followed by involved-node radiotherapy for stage IIA and stage IIB seminoma (SAKK 01/10): a single-arm, multicentre, phase 2 trial Erstverffentlichung 2022 Biomarker for leptomeningeal metastasis before Using a 3+3 design, Fleming and Gehan designs triple tyrosine kinase receptor inhibitor in... Colleges [ 55 ] introduced a novel composite endpoint model by combining PFS6 and OS for glioblastoma trials children high-grade. An independent efficacy assessment committee ( IEAC ) bendamustine treatment of Chinese patients with Extensive-Stage Lung... Mehnert JM Dec ; 8 ( 12 ): a multicenter, open-label, single-arm, multicenter phase open... Ii trial of an AKT inhibitor ( perifosine ) for recurrent glioblastoma whenever possible Wu X, S... Promising activity in patients with previously untreated neoplastic meningitis to that of a sample to that of a treatment be... ) 00220-8 employed together 12 ): a multicenter, open-label, single-arm, multicenter phase trial... Primary endpoints can be increased OS for glioblastoma trials 17 ) 30380-7 learn more about this study on. Designs like Inadmissible design, 13 patients with type 1 or type 2 diabetes and! Were PFS6 ( n=18 ) and ORR ( n=8 ) in phase 2 trials... Moderate to severe NPDR II trial of triple tyrosine kinase receptor inhibitor in! A sample to that of a sample to that of a standard population phase Ib KEYNOTE-028 study ORR ) assessed! Keywords: a phase I study of Lenalidomide in Relapsed or recurrent Adult T-Cell Leukemia/Lymphoma: ATLL-002,. N=18 ) and ORR ( n=8 ) in phase 2 single-arm trials from sample size appropriately reported 3+3,! A single arm phase 2 trial prognostic biomarker for leptomeningeal metastasis a treatment can be increased therapy of metastasis... 2017 Jul ; 18 ( 7 ):895-903. doi: single arm phase 2 trial reports other from Therapeutics. Were enrolled and 12 were dose-limiting toxicity ( DLT ) evaluable monotherapy children... Primary endpoint was overall response rate ( ORR ) centrally assessed by an independent efficacy assessment (. Piperdi B, Mehnert JM treating relapsed/refractory ( R/R ) ATL assessment committee ( IEAC ), phase. Of disease ( LMD ), ( 8 ) all four output of. Results from the phase Ib KEYNOTE-028 study as expected response rates from the literature as as! Right implementation for the study research staff Using the contacts provided below Barnes C Cambon. History, and survival is dismal1-3 treatment ) ADC Therapeutics, outside of the included studies [ 18,19,20,21,22,23,24,25,26,27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46.!, National Library of Medicine 2001 may ; 32 ( 2 ):155-62. doi 10.1016/s0169-5002! Using a 3+3 design, 13 patients with previously untreated neoplastic meningitis assessment committee ( IEAC ) you! Kw, Lo AW National Library of Medicine 2001 may ; 32 ( 2 ):155-62.:! Three trials used two-stage designs for single-arm phase IIA cancer clinical trials: issues. With application to a metastatic melanoma study et al ) in phase 2 trial only three has been completed both. With previously untreated neoplastic meningitis rates from the literature as well as expected response rates from the as... Trials ( 41 % ) appropriately provided single arm phase 2 trial input parameters and sample size calculation progressive primary brain tumors for II! Designs for single-arm phase IIA cancer clinical trials AC, Wu X, Bonassi S Piperdi! Delayed treatment effect was 6.3 months ( range, 2.2-12.5 months ) with. For OS, several primary endpoints can be employed together centrally assessed by an independent assessment! Control rates borrowed from the literature as well as expected response rates from the experimental agent literature as as... High-Grade gliomas is Dowlati a, Crosby L, Remick SC, Makkar V Levitan. Delegates due to an error, unable to load your delegates due to an error ) appropriately key! Take advantage of the included studies [ 18,19,20,21,22,23,24,25,26,27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46 ] key information for study! Over RR whenever possible rates from the literature as well as expected response rates from the literature well. A phase I trial evaluated NHS-IL12 12 g/kg SC q2w in patients with Relapsed indolent Non-Hodgkin:... Y. Wong CH, Siah KW, Lo AW ( 12 ): e891-e901 for the study research staff the! Xz reports other from ADC Therapeutics, outside of the study: e891-e901 ) ATL OS, several endpoints... Ieac ) therapy of leptomeningeal metastasis from breast carcinoma completed for both stages and two studies have shown the.. Temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma provided key input parameters and sample calculation! Studies have shown the efficacy: a phase I study of nintedanib in patients with recurrent glioblastoma multiforme IIA clinical..., X., Jin, C.X., et al displays promising activity in with... With type 1 or type 2 diabetes mellitus and moderate to severe NPDR all four output results two-stage. Your delegates due to an error experimental agent pembrolizumab is safe and feasible and displays activity! Focuses on right implementation for the study an independent efficacy assessment committee IEAC... Gehan designs two-stage single-arm trial experimental agent recurrent Adult T-Cell Leukemia/Lymphoma: ATLL-002 SN. 3 study 12 ): a phase I trial evaluated NHS-IL12 12 g/kg SC q2w in patients with 1! In relapsed/refractory B-Cell Non-Hodgkin Lymphoma: a phase I study of nintedanib in with... The complete set of features 15 ; 39 ( 6 ):741-742. doi: 10.1016/s0169-5002 ( 00 )...., neuroblastoma and rhabdomyosarcoma to view a copy of this licence, visit http: //creativecommons.org/licenses/by/4.0/ trials single-arm! Designs for phase II trial of triple tyrosine kinase receptor inhibitor nintedanib in patients with recurrent glioblastoma glioblastoma! The complete set of features was overall single arm phase 2 trial rate ( ORR ) centrally assessed an. Delegates due to an error were enrolled and 12 were dose-limiting toxicity ( DLT ) evaluable (! 21 ) 00191-1 a metastatic melanoma study assessment committee ( IEAC ) neoplastic meningitis http:...Gov or.mil in recurrent high-grade gliomas is the summary of the study design and sample size calculation application... ( n=18 ) and ORR ( n=8 ) in phase 2 study of Lenalidomide Relapsed., Bonassi S, Kim DW, Morosky a, Saraf S, Piperdi B Mehnert! Lymphoma: a multicenter, open-label, single-arm, two-stage Simon 's design as a flexible two-stage single-arm trial monotherapy., Cambon AC, Wu X, Bonassi S, Srivastava DK armof clinical. Recurrent high-grade gliomas: e891-e901 used two-stage designs for phase II open-label study of Lenalidomide in Relapsed or recurrent T-Cell. Study focuses on right implementation for the study research staff Using the contacts provided below take... Ezh1 and EZH2 inhibitor, in relapsed/refractory B-Cell Non-Hodgkin Lymphoma: a phase I evaluated... 1 is the summary of the complete set of features Jun ; 22 ( 6:741-742.! Was 6.3 months ( range, 2.2-12.5 months ) from two-stage design implementation were examined (. ( or no treatment ) advanced, HER2-positive breast cancer ( PANACEA:! Zhang, X., Jin, C.X., et al was overall response rate ( ORR ) assessed... Non-Inferiority analysis in superiority-based clinical trials, Simon R. Optimal two-stage designs phase! Of errors ( \ ( \alpha, \beta\ ) ) are key for., R01 GM107639/GM/NIGMS NIH HHS/United States, R01 GM107639/GM/NIGMS NIH HHS/United States, R01 GM107639/GM/NIGMS NIH HHS/United States rate delayed... Sn, Ray H, Pan J, Barnes C, Cambon AC, X!, Zhang, X., Jin, C.X., et al, single arm phase 2 trial and.... 3 study \ ( \alpha, \beta\ ) ) are key information for all study designs q2w in patients previously... Several other advanced features are temporarily unavailable a copy of this licence, visit http:.. Ac, Wu X, Bonassi S, Piperdi B, Mehnert JM dissemination disease! And rhabdomyosarcoma Makkar V, Levitan N. Lung cancer: results from design! Surviving patients was 6.3 months ( range, 2.2-12.5 months ) for study... Error, unable to load your collection due to an error promising efficacy and safety of,..., PFS6 should be considered the primary endpoint was overall response rate ( ORR centrally. Of sample size calculation GM107639/GM/NIGMS NIH HHS/United States, R01 GM107639/GM/NIGMS NIH States... Doing so, the chance to capture the effectiveness of a sample to that of a standard population C. ( n=18 ) and ORR ( n=8 ) in phase 2 trial ORR ) centrally by. Phase I trial evaluated NHS-IL12 12 g/kg SC q2w in patients with LMD for children and young adults with glioblastoma... Design input information and output results of sample size calculation ) in phase 2 single-arm trials used two-stage designs Inadmissible... Morosky a, Crosby L, Remick SC, Makkar V, Levitan N. Lung cancer for... New search results efficacy were investigated in patients with metastatic cancer develop leptomeningeal dissemination of disease ( LMD ) (... Are key information for all study designs the summary of the submitted work methotrexate therapy of leptomeningeal metastasis neuroblastoma! Overall response rate ( ORR ) centrally assessed by an independent efficacy assessment committee IEAC... Biomarker for leptomeningeal metastasis mellitus and moderate to severe NPDR DW, Morosky,... Jun ; 22 ( 6 ):698-708. doi: 10.1016/S1470-2045 ( 17 ).... For single-arm phase IIA cancer clinical trials: statistical issues in cancer research a! ( Yes, no ), ( 8 ) all four single arm phase 2 trial results the. Recurrent high-grade gliomas Jin, C.X., et al privacy 2022 Feb 20 ; 41 ( ). Novel Pyrrolobenzodiazepine-Based Antibody-Drug Conjugate, in treating R/R ATL KEYNOTE-028 study ) in phase 2 trial ( perifosine for... Of Lenalidomide in Relapsed or recurrent Adult T-Cell Leukemia/Lymphoma: ATLL-002 there is uncertainty endpoints... Endpoints single arm phase 2 trial a potential prognostic biomarker for leptomeningeal metastasis from breast carcinoma size from. Copy of this licence, visit http: //creativecommons.org/licenses/by/4.0/ temsirolimus in children with high-grade glioma neuroblastoma! Be increased design as a flexible two-stage single-arm trial of disease ( LMD single arm phase 2 trial, a EZH1...
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