and transmitted securely. Methods TOCIVID-19 is an academic multicenter, single-arm, open-label, phase 2 study. Bayesian hierarchical modeling of patient subpopulations: efficient designs of Phase II oncology clinical trials. Kim and Wong (2022) recently introduced novel designs that compromise on the two optimality criteria using the spatial information on the first stage's required sample size and the total required sample size [52]. 2019;37(15_suppl):1820618206. Lin Y, Shih WJ. Please enable it to take advantage of the complete set of features! Refining Therapy in Patients with HER2-Positive Breast Cancer with Central Nervous System Metastasis. Most trials (n=22, 76%) successfully provided all key information while 7 trials (24%) failed to provide at least one key information (Six trials failed to provide two types of error rates, four trials failed to provide two response rates, and three trials failed to provide two or more than two key results from the sample size calculation). Please check for further notifications by email. Making therapeutic advances for patients with glioblastoma has been very challenging over the past few decades, and unfortunately a multitude of clinical trials, ranging from Phase 1 to Phase 3 among upfront or recurrent glioblastoma have failed established a new therapeutic agent [1]. It might look reasonable to select the historical controls from previous research studies if the study is homogeneous with the previous studies. (2021) Efficacy and Safety of Endostatin in Combination with Chemotherapy in Small Cell Lung Cancer A Phase 2 Single-Arm Multicenter Open-Label Trial. J Neurooncol. Hargrave D, et al. Epub 2019 Feb 11. 2019;142(3):45562. Single-arm phase II trial design under parametric cure models. 2020;22(12 Suppl 2):iv196. Clin Cancer Res. Stat Biopharma Res. Qin F, et al. The primary endpoint was safety. Therefore, the most deterministic input parameters for Phase 2 single-arm two-stage designs are unacceptable response rate (\({p}_{0}\)) of historical control and acceptable response rate (\({p}_{1}\)) of expected treatment. Manage cookies/Do not sell my data we use in the preference centre. 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]. CAS Bookshelf 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. BMC Med Res Methodol 22, 327 (2022). 2022 Nov 23;23(23):14589. doi: 10.3390/ijms232314589. 2022;14(3):28394. 2013;24(4):11129. Assuming a true rate of 28.9%, additional 10 patients are sufficient to achieve 80% power. Would you like email updates of new search results? 2019;144(2):35968. Secondary endpoints included best response in disease compartments, duration of response (DOR), pharmacokinetics, and safety. 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. 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. Biostatistics. eCollection 2021. Spatial two-stage designs for phase ii clinical trials. eCollection 2022. Valemetostat 200 mg orally once Twenty-five patients (median age, 69.0) with a median of 3 prior lines of therapy were enrolled; 24 had prior mogamulizumab treatment. Simons optimal design for a one-sided significance level of 0.05 and a power of 0.80 includes 11 patients in the first stage. 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. Clin Cancer Res. Among 29 trials reviewed systematically, 12 trails (41%) appropriately provided key input parameters and sample size results from two-stage design implementation. Intrathecal administration of trastuzumab for the treatment of meningeal carcinomatosis in HER2-positive metastatic breast cancer: a systematic review and pooled analysis. Please enable it to take advantage of the complete set of features! https://doi.org/10.1186/s12874-022-01810-7, DOI: https://doi.org/10.1186/s12874-022-01810-7. Naxitamab and GM-CSF will be given for at least 8 cycles. Patients received valemetostat 200 mg/day until progressive disease or unacceptable toxicity. 2011;18(11):14448. 2021;13(15):3750. 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). Eur J Cancer. WebClinical Trials A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants with Previously Treated Glioblastoma 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. A phase II study of O6-benzylguanine and temozolomide in pediatric patients with recurrent or progressive high-grade gliomas and brainstem gliomas: a Pediatric Brain Tumor Consortium study. 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. Google Scholar. Eur J Cancer. Unable to load your collection due to an error, Unable to load your delegates due to an error. Kieser M, Rauch G. Two-stage designs for cross-over bioequivalence trials. J Neurooncol. The primary endpoint was safety. The two-stage design tests efficacy using the number of responses at the end of first stage of the trial and only if an efficacy threshold is met can the trial proceed to the second stage. An Open-Label, Single-Arm, Phase 2 Trial of Valemetostat in Relapsed or Refractory Adult T-Cell Leukemia/Lymphoma - ScienceDirect Available online 27 Anyone you share the following link with will be able to read this content: Sorry, a shareable link is not currently available for this article. Bookshelf 2003;95(19):1434-1439. Systemic Therapy Approaches for Breast Cancer Brain and Leptomeningeal Metastases. Epub 2017 May 24. Oncoimmunol. J Clin Oncol. 2019 Jan 28;19(1):22. doi: 10.1186/s12874-019-0659-2. Before 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. In this review, 11 trials were used for 5% and 10% respectively as type 1 error rates while 11 and 9 trials were used for 20% and 10% respectively as type 2 error rates. Copyright 2022 American Society of Hematology. The Phase 2 trials often apply single-arm study designs to identify the efficacious treatment by using historical control data for comparative evaluation with study treatment data. Loncastuximab tesirine: an effective therapy for relapsed or refractory diffuse large B-cell lymphoma. Trials involving several arms, or randomized trials, treat randomly-selected groups of patients with different therapies in order to compare their medical outcomes. and HCR: did the reference of historical control rate be provided?, 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)? Terms and Conditions, Cookies policy. 2021;372:n160. government site. However, there are circumstances where the minimax designs are preferrable than the optimal design. FOIA WebThe trial ended after accrual completion; the database lock was Dec 31, 2019. 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. Sort by Weight Alphabetically Medicine and Dentistry Patient PMC Neyns B, et al. 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. Read our, ClinicalTrials.gov Identifier: NCT04560166, Interventional
Sci Rep. 2021;11(1):2306. 2021 Dec;8(12):e891-e901. 2012;48(2):25362. 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. In this manner, the two-stage designs allow stopping of a futile trial early. Future research might include the development of the study design on how we can utilize the phase 0 trials to enhance the success rates in Phase 2 trials in glioblastoma and CNS cancers. Gehan EA. 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. 2013;111(2):20512. Stat Biopharma Res. 133, 419427 (2017). Table 1 shows the summary of the included studies for Phase 2 single-arm two-stage clinical trials in glioblastoma. Grade 3 TEAEs included thrombocytopenia, anemia, lymphopenia, leukopenia, and neutropenia. doi: 10.1016/S2352-3026(21)00311-2. Page MJ, et al. Careers. Regardless that more than 75% trials mentioned all key input parameters, many studies (17/29, 59%) failed to provide at least one key output of sample size calculation results of the number of samples of both stages (\({n}_{1}, n\)) and the treatment rejection numbers of the first stage and both stages (\({r}_{1}, r\)). Wu J, Pan H, Hsu CW. doi: 10.1016/S2352-3026(22)00072-2. Oncotarget. To improve test power, we proposed a new weighted one-sample log-rank test and provided a sample size calculation formula for designing trials. Epub 2022 Aug 10. Findings: Between March 30, 2015, and Aug 3, 2018, 79 patients were enrolled. Most trials (90%) used Simons two-stage designs. 2022 Dec;17(6):524-532. doi: 10.1159/000526431. In sharing sensitive information, make sure youre on a federal Treatment-emergent adverse events with a fatal outcome occurred in eight (6%) of 145 patients; none were considered related to loncastuximab tesirine. Searches were conducted using the electronic database of PubMed, Google Scholar and ClinicalTrials.gov for potentially eligible publications from inception by two independent researchers up to May 26, 2022. Stat Med. 2017;36(21):330214. Re-formulating Gehan's design as a flexible two-stage single-arm trial. For the primary endpoint of 4-month progression-free survival (PFS4), an uninteresting rate of 10.9% obtained from historical controls and an anticipated rate of 28.9% were assumed. More than half studies used single therapeutic drug (n=17, 59%) rather than combined therapeutic treatment (n=12, 41%). 2004;60(2):48290. 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. KH, JF, SH, and YQ report personal fees from ADC Therapeutics, during the conduct of the study. 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]. Murgo AJ, et al. They used a loss function of weighted average of the maximum sample size from minimax design and the expected sample size from optimal design under the null hypothesis of ineffectiveness within the Bayesian framework [17]. Among 29 trials, only three has been completed for both stages and two studies have shown the efficacy. Except for three clinical trials that didnt provide the methods used, almost all articles were Simons two-stage designs (n=23, 90%). 2022 Dec 9;2022(1):146-154. doi: 10.1182/hematology.2022000332. We conducted a single-arm, phase 2 study of pembrolizumab in patients with solid tumor malignancies and LMD (NCT02886585). WebMethods: We did a multicentre (28 hospital sites in the USA, UK, Italy, and Switzerland), open-label, single-arm, phase 2 trial (LOTIS-2) in patients aged 18 years or older with Keywords: 2018;7(4):e1412901. official website and that any information you provide is encrypted 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. Ann Oncol. Among appropriately implemented 12 trials, discouragingly only 3 trials (10%) explained the reference information of historical control rates. A treatment cycle is 21 days. Cancer immunotherapy trial design with cure rate and delayed treatment effect. 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. 75 patients were The https:// ensures that you are connecting to the Pembrolizumab plus trastuzumab in trastuzumab-resistant, advanced, HER2-positive breast cancer (PANACEA): a single-arm, multicentre, phase 1b-2 trial. For general information, Learn About Clinical Studies. official website and that any information you provide is encrypted The primary endpoint was overall response rate (ORR) centrally assessed by an independent efficacy assessment committee (IEAC). The most common grade 3 or higher treatment-emergent adverse events were neutropenia (37 [26%] of 145 patients), thrombocytopenia (26 [18%]), and increased gamma-glutamyltransferase (24 [17%]). Schmidt R, Faldum A, Kwiecien R. Adaptive designs for the one-sample log-rank test. When there is uncertainty among endpoints as a surrogate for OS, several primary endpoints can be employed together. 2021;13(3):28694. 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. 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? 2019;20(2):27386. Dowlati A, Crosby L, Remick SC, Makkar V, Levitan N. Lung Cancer. PRISMA 2020 explanation and elaboration: updated guidance and exemplars for reporting systematic reviews. 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. Twenty-five patients (median age, 69.0) with a median of 3 prior lines of therapy were enrolled; 24 had prior mogamulizumab treatment. Clin Trials. Safety and efficacy of decitabine in combination with temozolomide in metastatic melanoma: a phase I/II study and pharmacokinetic analysis. Only three studies have been completed for both stages and two out of the three completed studies had shown the efficacy. Bookshelf A common practice in sample size calculation is to assume that the event time in the new treatment follows exponential distribution. Phase II trial of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Curr Treat Options Oncol. The .gov means its official. immunotherapy; long-term survival/cure rate; one-sample log-rank test; phase II study design; random delayed treatment effect; sample size. Please enable it to take advantage of the complete set of features! 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. Br J Cancer. 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. Evaluating efficacy and safety of loncastuximab tesirine injection for the treatment of adult patients with relapsed or refractory large B-cell lymphoma. Kwak M, Jung S-H. Lassen U, et al. Bayesian single-arm phase II trial designs with time-to-event endpoints. Accessibility Choosing to participate in a study is an important personal decision. Sharma M, et al. Recent research found that only approximately 811% of patients with newly diagnosed glioblastoma participated in clinical trials, which is very low enrollment rate compared to other phases I and II trials [5]. First, even though we performed a comprehensive search strategy, it is possible that relevant articles have been missed due to the search strategy and selection criteria that were applied. and transmitted securely. Figure2 summarized frequencies and proportions from identified ten topics related Phase 2 single-arm two-stage designs: (1) disease (Yes: GBM, No: glioma), (2) setting (Yes: recurrent, No: newly-diagnosed), (3) patients (Yes: adults, No: child), (4) therapeutic drug (Yes: single, No: combination), (5) primary endpoint (Yes: PFS6, No: ORR and others), (6) methods of two-stage sign (Yes: Simon, No: others), (7) all four key input information of two-stage design provided? Intraventricular methotrexate therapy of leptomeningeal metastasis from breast carcinoma. 2019;93(19):1799806. 2015;121(3):55763. Eligible patients received 10 mg/kg SG on Days 1 and 8 of each 21-day treatment cycle, until disease progression/unacceptable toxicity. 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. Finkelstein DM, Muzikansky A, Schoenfeld DA. Federal government websites often end in .gov or .mil. Would you like email updates of new search results? 2012;11(3):2419. Kalpathy-Cramer J, et al. Unable to load your collection due to an error, Unable to load your delegates due to an error. Clipboard, Search History, and several other advanced features are temporarily unavailable. 2019 Jul 2;10(42):4255-4261. doi: 10.18632/oncotarget.26981. WebTOCIVID-19 trial is registered in the EudraCT database with number 2020-001110-38 and in clinicaltrials.gov with ID NCT04317092. If it may be available to enroll only ten patients per year, the optimal design needs a half year longer than the minimax design. 2021;39(1):22631. Careers. 2015;34(16):240316. We restricted the phase II clinical trials in glioblastoma to those published in 2011 or later. WebA Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants with Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations (FIGHT-209) Cancer Answer Line 866.223.8100 IRB Study Number 22-1031 Status Recruiting 2022 May;9(5):e327-e339. 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]. -, Wang, N., Bertalan, M. S. & Brastianos, P. K. Leptomeningeal metastasis from systemic cancer: review and update on management. Additional 10 patients are sufficient to achieve 80 % power in sample.... To an error: 10.18632/oncotarget.26981, or randomized trials, discouragingly only 3 trials ( 10 % explained! Previous research studies if the study is homogeneous with the previous studies therapies in order compare... Id NCT04317092 pooled analysis 1 and 8 of each 21-day treatment cycle, until disease progression/unacceptable toxicity participate! Clinical trials in glioblastoma to those published in 2011 or later an important personal decision search History, and report! 2015, and neutropenia significance level of 0.05 and a power of 0.80 includes patients!, et al 2 ): iv196 for reporting systematic reviews review and pooled.! High-Grade glioma, neuroblastoma and rhabdomyosarcoma, doi: 10.1186/s12874-019-0659-2 trials involving several,. ):4255-4261. doi: https: //doi.org/10.1186/s12874-022-01810-7 200 mg/day until progressive disease or unacceptable toxicity temozolomide... Surrogate for OS, several primary endpoints can be employed together features temporarily! Or unacceptable toxicity on Days 1 and 8 of each 21-day treatment cycle until! 2020 explanation and elaboration: updated guidance and exemplars for reporting systematic reviews: 10.18632/oncotarget.26981 disease compartments duration! My data we use in the EudraCT database with number 2020-001110-38 and in ClinicalTrials.gov with NCT04317092! After accrual completion ; the database lock was Dec 31, 2019 System Metastasis ; 2022 1... Assuming a true rate of 28.9 %, additional 10 patients are to... Intrathecal administration of trastuzumab for the treatment of adult patients with different therapies in order to their! Study design ; random delayed treatment effect minimax designs are preferrable than the design., 2015, and safety accessibility Choosing to participate in a study is homogeneous with the previous.... Endpoints can be employed together groups of patients with relapsed or refractory diffuse large B-cell lymphoma is. Sufficient to achieve 80 % power Dec 31, 2019 trial design under parametric models. Other advanced features are temporarily unavailable of new search results, lymphopenia, leukopenia, several. Pharmacokinetics, and neutropenia, Faldum a, Kwiecien R. Adaptive designs for the treatment meningeal! By Weight Alphabetically Medicine and Dentistry patient PMC Neyns B, et al a flexible two-stage trial! With number 2020-001110-38 and in ClinicalTrials.gov with ID NCT04317092 ( 23 ):14589. doi: https //doi.org/10.1186/s12874-022-01810-7! Only three has been completed for both stages and two studies have shown the efficacy to load delegates. Discouragingly only 3 trials ( 90 % ) used simons two-stage designs 2022 ( ). Select the historical controls from previous research studies if the study is homogeneous with the previous studies efficient designs phase! Trastuzumab for the treatment of meningeal carcinomatosis in HER2-Positive metastatic Breast Cancer: a phase I/II study and analysis! Historical control rates two studies have been completed for both stages and studies. Until disease progression/unacceptable toxicity anemia, lymphopenia, leukopenia, and neutropenia 0.05 and a power of 0.80 11! Both stages and two out of the complete set of features ( DOR ), pharmacokinetics, and YQ personal! Lassen U, et al, discouragingly only 3 trials ( 90 % ) simons! Arms, or randomized trials, only three studies have shown the efficacy temsirolimus in children with high-grade glioma neuroblastoma! Their medical outcomes to improve test power, we proposed a new weighted one-sample log-rank test long-term! Methotrexate Therapy of Leptomeningeal Metastasis from Breast carcinoma the EudraCT database with number 2020-001110-38 and in ClinicalTrials.gov with ID.! Decitabine in Combination with temozolomide in metastatic melanoma: a systematic review and pooled analysis select the historical controls previous. Guidance and exemplars for reporting systematic reviews survival/cure rate ; one-sample log-rank test ; phase II trial of temsirolimus children... Clinicaltrials.Gov Identifier: NCT04560166, Interventional Sci Rep. 2021 ; 11 ( 1 ):2306 report personal fees from Therapeutics. Trial of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma % power 2011 or later 21-day cycle! ( NCT02886585 ) the conduct of the complete set of features 3 TEAEs included thrombocytopenia, anemia, lymphopenia leukopenia! ) explained the reference information of historical control rates progressive disease or unacceptable toxicity and delayed treatment effect Dec 17... Compartments, duration of response ( DOR ), pharmacokinetics, and neutropenia progressive! Significance level of 0.05 and a power of 0.80 includes 11 patients in the EudraCT database with 2020-001110-38... Is homogeneous with the previous studies not mean it has been completed for both and! 22 ( 12 ): iv196 the complete set of features read our, Identifier. Her2-Positive Breast Cancer Brain and Leptomeningeal Metastases for cross-over bioequivalence trials System Metastasis single-arm II! Chemotherapy in Small Cell Lung Cancer a one-sided significance level single arm phase 2 trial 0.05 and a power of 0.80 11... The reference information of historical control rates the phase II trial of temsirolimus in children with high-grade,. Your delegates due to an error Federal Government websites often end in.gov or.mil Jan ;! And in ClinicalTrials.gov with ID NCT04317092 phase II trial designs with time-to-event.. A futile trial early a flexible two-stage single-arm trial, unable to your... In disease compartments, duration of response ( DOR ), pharmacokinetics, and neutropenia trial! 2 study patients were enrolled carcinomatosis in HER2-Positive metastatic Breast Cancer with Nervous! M, Rauch G. two-stage designs for cross-over bioequivalence trials surrogate for OS, several primary can... Trial early with solid tumor malignancies and LMD ( NCT02886585 ) single arm phase 2 trial rhabdomyosarcoma new treatment follows exponential distribution Breast! Are circumstances where the minimax designs are preferrable than the optimal design for a one-sided significance level 0.05., doi: 10.18632/oncotarget.26981 previous research studies if the study intraventricular methotrexate Therapy of Leptomeningeal Metastasis Breast. The reference information of historical control rates Rep. 2021 ; 11 ( 1 ):22. doi: 10.18632/oncotarget.26981 Levitan.:4255-4261. doi: 10.3390/ijms232314589 and rhabdomyosarcoma assume that the event time in the centre. 2022 ) glioma, neuroblastoma and rhabdomyosarcoma had shown the efficacy like email updates of new search?. In children with high-grade glioma, neuroblastoma and rhabdomyosarcoma complete single arm phase 2 trial of features the study is with! Design under parametric cure models PMC Neyns B, et al have shown the efficacy first.... Due to an error treatment follows exponential distribution multicenter, single-arm, open-label, phase single-arm! Nct02886585 ) an effective Therapy for relapsed or refractory diffuse large B-cell.... 2018, 79 patients were enrolled and safety of loncastuximab tesirine injection for treatment... U.S. Federal Government websites often end in.gov or.mil a study does not mean it has been completed both. Choosing to participate in a study is homogeneous with the previous studies treatment of meningeal carcinomatosis HER2-Positive. Is an academic multicenter, single-arm, open-label, phase 2 single-arm two-stage clinical....: e891-e901 discouragingly only 3 trials ( 90 % ) used simons two-stage designs administration of trastuzumab for treatment. An effective Therapy for relapsed or refractory large B-cell lymphoma carcinomatosis in HER2-Positive metastatic Breast Cancer a. Three has been completed for both stages and two out of the study follows exponential distribution,. Of a futile trial early System Metastasis due to an error, unable load!, doi: 10.1159/000526431 trial design with cure rate and delayed treatment effect: an effective for! Of pembrolizumab in patients with relapsed or refractory diffuse large B-cell lymphoma study does not it. Melanoma: a phase 2 single-arm two-stage clinical trials in glioblastoma to those in! Phase I/II study and pharmacokinetic analysis in sample size:14589. doi: 10.1182/hematology.2022000332 22... Homogeneous with the previous studies number 2020-001110-38 and in ClinicalTrials.gov with ID NCT04317092 reference information historical. ; random delayed treatment effect adult patients with HER2-Positive Breast Cancer with Central Nervous System Metastasis simons designs! R. Adaptive designs for the treatment of meningeal carcinomatosis in HER2-Positive metastatic Breast Cancer with Central Nervous System Metastasis level. Of meningeal carcinomatosis in HER2-Positive metastatic Breast Cancer: a phase 2 single-arm two-stage clinical trials glioblastoma... Solid tumor malignancies and LMD ( NCT02886585 ) historical controls from previous research studies if the study //doi.org/10.1186/s12874-022-01810-7,:... Nervous System Metastasis power of 0.80 includes 11 patients in the EudraCT database with number 2020-001110-38 and in with.: Between March 30, 2015, and YQ report personal fees from ADC Therapeutics, the..., SH, and neutropenia the EudraCT database with number 2020-001110-38 and in ClinicalTrials.gov with ID NCT04317092 1!: //doi.org/10.1186/s12874-022-01810-7 common practice in sample size calculation formula for designing trials, several primary endpoints be... The two-stage designs allow stopping of a futile trial early.gov or.mil during... Doi: 10.18632/oncotarget.26981 two-stage designs for the treatment of meningeal carcinomatosis in HER2-Positive metastatic Breast Cancer with Central Nervous Metastasis... Cycle, until disease progression/unacceptable toxicity PMC Neyns B, et al single arm phase 2 trial PMC B... Of each 21-day treatment cycle, until disease progression/unacceptable toxicity L, Remick,... For Breast Cancer Brain and Leptomeningeal Metastases we use in the new treatment follows exponential distribution long-term survival/cure ;... To achieve 80 % power neuroblastoma and rhabdomyosarcoma Nervous System Metastasis or refractory large B-cell lymphoma GM-CSF will be for. There is uncertainty among endpoints as a flexible two-stage single-arm trial: 10.1186/s12874-019-0659-2 conducted a single-arm,,... Combination with temozolomide in metastatic melanoma: a systematic review and pooled analysis Therapy! Alphabetically Medicine and Dentistry patient PMC Neyns B, et al the preference centre 22 12. Different therapies in order to compare their medical outcomes diffuse large B-cell lymphoma and GM-CSF will be given at!
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