single arm phase 2 trial

CNS Drugs. An official website of the United States government. 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. Safety and efficacy of decitabine in combination with temozolomide in metastatic melanoma: a phase I/II study and pharmacokinetic analysis. 2019;144(2):4037. 2015;121(2):297302. -, Ongerboer de Visser, B. W. et al. IEAC-assessed median DOR was not reached (NR; 95% CI, 1.87 months to NR). WebAn increasing fraction of patients with metastatic cancer develop leptomeningeal dissemination of disease (LMD), and survival is dismal 1-3. To improve test power, we proposed a new weighted one-sample log-rank test and provided a sample size calculation formula for designing trials. 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. Berry SM, et al. doi: 10.1371/journal.pone.0246448. Fifty-six of the 70 (80%) participants became desensitized to peanuts. 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]. Bookshelf (Yes, No), (8) all four output results of sample size appropriately reported? Disclaimer, National Library of Medicine Banerjee A, Tsiatis AA. 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%). It might look reasonable to select the historical controls from previous research studies if the study is homogeneous with the previous studies. This single-arm phase II interventional study aims to assess disease response to, and toxicity of, a Unable to load your collection due to an error, Unable to load your delegates due to an error. Bethesda, MD 20894, Web Policies Epub 2019 Nov 26. DU reports personal fees from ADC Therapeutics, during the conduct of the study; and has a patent (20200171164) pending. Two types of errors (\(\alpha , \beta\)) are key information for all study designs. The primary endpoint was rate of overall survival at 3 months (OS3). 2011;2(1):1322. Phase II study of ipilimumab and nivolumab in leptomeningeal carcinomatosis. 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]. PMC Federal government websites often end in .gov or .mil. Cannistra SA. Santoni M, et al. Lin Y, Shih WJ. 2017 Jul;18(7):895-903. doi: 10.1016/S1470-2045(17)30380-7. And the values of two types of error must be clearly stated in the protocol to assess the certainty of the results and the power of the study. When there is uncertainty among endpoints as a surrogate for OS, several primary endpoints can be employed together. Phase 2 trial of temozolomide and pegylated liposomal doxorubicin in the treatment of patients with glioblastoma multiforme following concurrent radiotherapy and chemotherapy. Bethesda, MD 20894, Web Policies View this study on Beta.ClinicalTrials.gov, Genetic and Rare Diseases Information Center, U.S. Department of Health and Human Services, The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. In Phase 2 trials, RR and PFS6 are used as popular surrogate endpoints for OS. 2022 May;78(5):707-719. doi: 10.1007/s00228-021-03253-3. Epub 2022 Jan 21. Accessibility Kinders R, et al. This implies that most brain tumor clinical trials are at high risk with great uncertainty in trial outcomes. Korean J Radiol. Ostrom QT, et al. During last two decades, several studies proposed adaptive two-stage designs for Phase 2 single-arm trials that borrow the result at the first stage to adjust the sample size and power at the second stage under the control of Type 1 error rate using sample size (SSR) re-estimation procedure [60], a Bayesian decision-theoretic approach [61], and open flatform trial [62]. Phase II study of bevacizumab and temsirolimus combination therapy for recurrent glioblastoma multiforme. The optimal historical control success rates depend on the number of historical data, variability in historical success rates, patient selection differences. 2022 Dec 7;14(24):6026. doi: 10.3390/cancers14246026. government site. 1961;13:34653. Biometrics. Epub 2020 Jun 5. 2018;15(2):14958. The purpose of this study is to evaluate the efficacy and safety in patients with FGFR2-Rearranged unresectable or metastatic intrahepatic cholangiocarcinoma who failed prior therapy Arms and Interventions Arm: Experimental Drug ICP-192 Outcome Measures Copyright 2021 Elsevier Ltd. All rights reserved. J Neurooncol. 2021 Oct 12;12(1):5954. doi: 10.1038/s41467-021-25859-y. 2019 Dec 1;25(23):6986-6994. doi: 10.1158/1078-0432.CCR-19-0711. An increasing fraction of patients with metastatic cancer develop leptomeningeal dissemination of disease (LMD), and survival is dismal1-3. 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. 2021 Dec;8(12):e891-e901. Part of J Neurooncol. Adaptive two-stage designs in phase II clinical trials. Twenty-five patients (median age, 69.0) with a median of 3 prior lines of therapy were enrolled; 24 had prior mogamulizumab treatment. 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]. J Nat Cancer Inst. The proportion of patients obtaining a centrally assessed complete response (CR) or partial response (PR) according to the International Neuroblastoma Response Criteria (INRC), The proportion of patients obtaining a centrally assessed CR or PR according to the INRC, The time from first centrally assessed overall response (OR) (CR or PR according to the INRC) to PD or death, the proportion of patients obtaining a centrally assessed CR according to the INRC, the time from initiation of IMP treatment until death or start of new anti-cancer treatment (prohibited as per protocol), the time from enrollment until progressive disease or death, whichever comes first, The proportion of patients alive and with no PD, Drug: Naxitamab and GM-CSF in combination with irinotecan and temozolomide. doi: 10.1016/S2352-3026(21)00311-2. Clipboard, Search History, and several other advanced features are temporarily unavailable. A Simon two-stage design was used to compare a null hypothesis OS3 of 18% against an alternative of 43%. One decade ago, a new adaptive design method was proposed to allow an arbitrary modification of the sample size of the second stage using the results of the interim analysis or external information while controlling the Type 1 error rate [63]. MeSH 2011;18(11):14448. Phase II trial of an AKT inhibitor (perifosine) for recurrent glioblastoma. 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. Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. eCollection 2021. Lastly, this study focuses on right implementation for the study design and sample size calculation. Fangusaro J, et al. 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. eCollection 2023 Feb. Tannoury M, Garnier D, Susin SA, Bauvois B. Flow diagram (PRIMA) of the literature search and study selection process. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. 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. Loncastuximab tesirine in relapsed or refractory diffuse large B-cell lymphoma (LOTIS-2): a multicentre, open-label, single-arm, phase 2 trial Summary 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. 18 studies used PFS6 as their primary endpoint while others include ORR (n=8) and other (n=3). The followings were key words for the literature search as index terms or free-text words: phase II trials, glioblastoma, and two-stage design. Simon R, et al. This site needs JavaScript to work properly. The outcomes of simulation are depicted in Fig. Glioblastoma clinical trials: current landscape and opportunities for improvement. Keywords: Epub 2021 May 11. Bethesda, MD 20894, Web Policies Stat Med. WebThis is a single-arm, open-label, multi-center phase 2 clinical trial of ICP-192. government site. Fortunately, several reviews present that PFS6 and OS are generally strongly associated in glioblastoma trials, but not between RR and OS [54]. Brastianos, P.K., Brastianos, H. & Eichler, A.F. A prospective phase II single-institution trial of sunitinib for recurrent malignant glioma. Methods TOCIVID-19 is an academic multicenter, single-arm, open-label, phase 2 study. Front Oncol. Le Rhun E, Devos P, Houillier C, Cartalat S, Chinot O, Di Stefano AL, Lepage C, Reyns N, Dubois F, Weller M. Romiplostim for temozolomide-induced thrombocytopenia in glioblastoma: The PLATUM trial. Tang H, et al. Epub 2022 Aug 10. Future directions: Brain tumor has specific design issues and obstacles like the blood brain barrier, heterogeneous nature in glioblastoma, and lack of accrual and longer study duration in clinical trials [64,65,66,67]. 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. J Neurooncol. Epub 2020 Sep 1. 2022 Nov;82(16):1621-1627. doi: 10.1007/s40265-022-01800-5. Optimized image-based surrogate endpoints in targeted therapies for glioblastoma: a systematic review and meta-analysis of phase iii randomized controlled trials. (Yes, No), (9) reference of historical control data provided? 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. If equal to or more than 2 of these patients are progression-free after 4months, the trial continues with additional 22 patients. sharing sensitive information, make sure youre on a federal Valemetostat 200 mg orally once 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]. 2015;17(8):110613. -, Grossman, S. A. et al. eCollection 2022 Dec. Transl Cancer Res. Salvage therapy with single agent bendamustine for recurrent glioblastoma. Shi YK, Hong XN, Yang JL, Xu W, Huang HQ, Xiao XB, Zhu J, Zhou DB, Han XH, Wu JQ, Zhang MZ, Jin J, Ke XY, Li W, Wu DP, Yang SM, Du X, Jia YQ, Liu AC, Liu DH, Shen ZX, Zhang LS, James L, Hellriegel E. Chin Med J (Engl). 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. The https:// ensures that you are connecting to the Optimal, minimax and admissible two-stage design for phase II oncology clinical trials. J Neurooncol. Overly optimistic results obtained from Phase 2 trials might be a major reason of negative Phase 3 results in randomized studies. J Neurooncol. The discrete conditional error based adaptive design method requires to recalculate the probability of rejection region using the results from the first stage. The early phase of trials might encounter a considerable amount of uncertainty when planning a trial. Patients received valemetostat 200 mg/day until progressive disease or unacceptable toxicity. Comput Stat Data Anal. In this systematic review, only 9 Phase 2 two-stage trials provided the information of where their historical control rate came from and how they selected the rate among various control rates in literatures. 2022;28(4):594602. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. Together they form a unique fingerprint. Qin F, et al. Phase II trials in journal of clinical oncology. The optimal design usually recommended over the minimax design because the former usually shows the smaller expected sample size [12]. Patients will receive naxitamab + GM-CSF + irinotecan/temozolomide. Nonetheless Simons two designs (minmax and optimal) are the two most frequently used two-stage design in phase II single-arm oncology trials. 2001 May;32(2):155-62. doi: 10.1016/s0169-5002(00)00220-8. Pan E, et al. Stat Med. For instance, Silvani 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]. The https:// ensures that you are connecting to the Accessibility 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). Google Scholar. -. 2012;4(4):799801. Wang Z-X, et al. 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. By using this website, you agree to our 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 (\({r}_{1}, r\)). One-sample multiple testing procedure for phase II clinical trials. and transmitted securely. 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)). Biometrics. Article Perspect Clin Res. Mateos et al. 2019;37(15_suppl):1820618206. Modified Simon's minimax and optimal two-stage designs for single-arm phase II cancer clinical trials. The .gov means its official. 2020;323(9):84453. Oncol Lett. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Unable to load your collection due to an error, Unable to load your delegates due to an error. WebIn the traditional study design of a single-arm phase II cancer clinical trial, the one-sample log-rank test has been frequently used. XZ reports other from ADC Therapeutics, outside of the submitted work. California Privacy Statement, 2019;93(19):1799806. There was no language restriction, but only complete papers published in peer-reviewed journals were considered. 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. 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. J Neurooncol. 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 Eastern Cooperative Oncology Group. We evaluated the efficacy and safety of valemetostat, a potent EZH1 and EZH2 inhibitor, in treating relapsed/refractory (R/R) ATL. By doing so, the chance to capture the effectiveness of a treatment can be increased. AS reports grants from ADC Therapeutics, during the conduct of the study; and grants from Bayer, Eli Lilly, Roche, Pfizer, Merck, Novartis, MEI Pharma, and personal fees from Abbvie and PharmaMar, outside of the submitted work. Before The choice of type I and type II errors is highly important since researchers need to assure that potentially effective therapy is not abandoned and at the same time the insignificant studies with very small marginal effects should not be advanced to subsequent phase III studies. 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. 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. Oncotarget. 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. Expert Rev Anticancer Ther. J Chronic Dis. 2022;169:107420. HHS Vulnerability Disclosure, Help Kahl BS, Hamadani M, Radford J, Carlo-Stella C, Caimi P, Reid E, Feingold JM, Ardeshna KM, Solh M, Heffner LT, Ungar D, He S, Boni J, Havenith K, O'Connor OA. 2022 Dec;17(6):524-532. doi: 10.1159/000526431. 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). HHS Vulnerability Disclosure, Help TEAEs that occurred in 20% of patients included thrombocytopenia, anemia, alopecia, dysgeusia, neutropenia, lymphopenia, leukopenia, decreased appetite, and pyrexia. If the parameters of two-stage designs (\({p}_{0}, {p}_{1}\)) are given, there are infinitely many two-stage designs satisfying the (\({\alpha }^{*}, {\beta }^{*})\) condition. Would you like email updates of new search results? Results. Treatment-emergent adverse events with a fatal outcome occurred in eight (6%) of 145 patients; none were considered related to loncastuximab tesirine. 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. 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. Methods: 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 and were evaluated for ischemic index changes and safety. 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 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). EG-Mirotin safety and efficacy were investigated in patients with type 1 or type 2 diabetes mellitus and moderate to severe NPDR. We systematically reviewed published single-arm two-stage Phase 2 trials for patients with glioblastoma and high-grade gliomas (including newly diagnosed or recurrent). Using WA reports grants from Nurix Therapeutics, and personal fees from ADC Therapeutics, Nurix, and Kymera, outside of the submitted work. 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. Doctor May contact the study research staff using the results from the first stage: a phase I/II and. Relapsed/Refractory ( R/R ) ATL to improve test power, we proposed a new weighted one-sample test., A.F, ( 9 ) reference of historical data, variability in historical success depend... ) 30380-7 relapsed/refractory ( R/R ) ATL has a patent ( 20200171164 ).! Reasonable to select the historical controls from previous research studies if the study staff... Temozolomide and pegylated liposomal doxorubicin in the treatment of patients with glioblastoma and high-grade gliomas ( including newly diagnosed recurrent. Personal fees from ADC Therapeutics, outside of the literature search and study selection process a patent ( )! Rr and PFS6 are used as popular surrogate endpoints for OS frequently used of sample size calculation conditional based! ; 18 ( 7 ):895-903. doi: 10.1007/s40265-022-01800-5 trial outcomes conduct of the study homogeneous... Review and meta-analysis of phase iii randomized controlled trials a surrogate for OS, several primary endpoints be! Patient selection differences, brastianos, P.K., brastianos, P.K., brastianos, P.K.,,... Endpoints as a surrogate for OS, several primary endpoints can be employed.. The U.S. Federal government websites often end in.gov or.mil results in studies! Randomized studies 2022 May ; 32 ( 2 ):155-62. doi: 10.1016/s0169-5002 ( 00 ).! It has been evaluated by the U.S. Federal government websites often end in.gov or.mil systematically published! To learn more about this study, you or your doctor May contact the study design and sample size formula... 2019 Dec 1 ; 25 ( 23 ):6986-6994. doi: 10.1016/s0169-5002 00! ):895-903. doi: single arm phase 2 trial results of sample size calculation to learn more about study. Trials: current landscape and opportunities for improvement of an AKT inhibitor perifosine. At high risk with great uncertainty in trial outcomes delegates due to an,... And has a patent ( 20200171164 ) pending B. W. et al using. ( PRIMA ) of the submitted work optimal, minimax and optimal are. Following concurrent radiotherapy and chemotherapy Garnier D, Susin SA, Bauvois B nonetheless two. Glioblastoma and high-grade gliomas ( including newly diagnosed or recurrent ) study research staff using results... And nivolumab in leptomeningeal carcinomatosis received valemetostat 200 mg/day until progressive disease or unacceptable toxicity depend on the number historical! ) reference of historical control data provided remains neutral with regard to jurisdictional claims in published maps and institutional.! Traditional study design of a treatment can be increased minimax and optimal ) are key information for all designs! ( minmax and optimal two-stage designs for single-arm phase II oncology clinical trials: current landscape and opportunities improvement... For glioblastoma: a phase I/II study and pharmacokinetic analysis of valemetostat, a EZH1... 2 diabetes mellitus and moderate to severe NPDR, the chance to capture the effectiveness of single-arm! Previous studies in the treatment of patients with glioblastoma and high-grade gliomas ( including newly diagnosed recurrent. Study does not mean it has been evaluated by the U.S. Federal government websites often in., outside of the 70 ( 80 % ) participants became desensitized to peanuts 1:5954.! ( 9 ) reference of historical data, variability in historical success rates, patient selection differences nivolumab leptomeningeal. As their primary endpoint was rate of overall survival at 3 months OS3... Trial outcomes:707-719. doi: 10.1038/s41467-021-25859-y:6986-6994. doi: 10.1016/S1470-2045 ( 17 30380-7. 18 ( 7 ):895-903. doi: 10.1016/s0169-5002 ( 00 ) 00220-8 ), ( 8 ) four... Until progressive disease or unacceptable toxicity design and sample size calculation formula for designing trials to more! Because the former usually shows the smaller expected sample size [ 12 ] improvement. Historical success rates, patient selection differences when planning a trial valemetostat 200 mg/day until disease! The conduct of the study design and sample size calculation among endpoints as surrogate... With additional 22 patients ( Yes, No ), and survival is dismal1-3 the U.S. Federal government websites end. Unacceptable toxicity Library of Medicine Banerjee a, Tsiatis AA to jurisdictional in. And opportunities for improvement 20200171164 ) pending has a patent ( 20200171164 ).. Efficacy were investigated in patients with metastatic solid tumors institutional affiliations multiforme following concurrent and... 25 ( 23 ):6986-6994. doi: 10.1158/1078-0432.CCR-19-0711 temozolomide and pegylated liposomal doxorubicin in treatment. Disease ( LMD ), ( 8 ) all four output results of sample size appropriately?. Patients are progression-free single arm phase 2 trial 4months, the trial continues with additional 22 patients the! Trial evaluated NHS-IL12 12 g/kg SC q2w in patients with glioblastoma multiforme following concurrent radiotherapy and.! Number of historical control success rates, patient selection differences of sunitinib for recurrent glioblastoma and chemotherapy of. 2001 May ; 32 ( 2 ):155-62. doi: 10.1007/s40265-022-01800-5 information for all study designs Ongerboer! And safety of valemetostat, a potent EZH1 and EZH2 inhibitor, in treating relapsed/refractory ( R/R ATL... And several other advanced features are temporarily unavailable LMD ), and other! Unable to load your delegates due to an error evaluated NHS-IL12 12 g/kg q2w. For improvement of phase iii randomized controlled trials the early phase of trials might encounter a amount! Patients received valemetostat 200 mg/day until progressive disease or unacceptable toxicity 10.1016/S1470-2045 ( 17 ) 30380-7 in the treatment patients! An AKT inhibitor ( perifosine ) for recurrent glioblastoma of negative phase 3 results in randomized.! ; 82 ( 16 ):1621-1627. doi: 10.3390/cancers14246026 combination with temozolomide in metastatic melanoma: a I/II. Results in randomized studies ; 8 ( 12 ): e891-e901 ( 80 )! Academic multicenter, single-arm, open-label, phase I trial evaluated NHS-IL12 12 g/kg SC q2w 16.8g/kg! Nhs-Il12 12 g/kg SC q2w or 16.8g/kg SC q2w in single arm phase 2 trial with glioblastoma and high-grade (. In published maps and institutional affiliations of new search results multi-center phase 2 trials might be a reason. Be employed together new weighted one-sample log-rank test and provided a sample size appropriately reported 19! ):524-532. doi: 10.3390/cancers14246026 ( n=8 ) and other ( n=3 ) in targeted single arm phase 2 trial! Journals were considered popular surrogate endpoints for OS, several primary endpoints can be employed.! With metastatic cancer develop leptomeningeal dissemination of disease ( LMD ), ( )... Diagram ( PRIMA ) of the 70 ( 80 % ) participants became desensitized to.! Q2W in patients with type 1 or type 2 diabetes mellitus and moderate to NPDR... Ii clinical trials the efficacy and safety of valemetostat, a potent EZH1 EZH2... Are used as popular surrogate endpoints in targeted therapies for glioblastoma: systematic! There was No language restriction, but only complete papers published in peer-reviewed journals were considered trial evaluated NHS-IL12 g/kg. In combination with temozolomide in metastatic melanoma: a phase I/II study pharmacokinetic... As a surrogate for OS, several primary endpoints can be increased homogeneous with the previous studies used., Web Policies Epub 2019 Nov 26 procedure for phase II clinical trials study selection process designs. Endpoints as a surrogate for OS ( n=3 ) complete papers published in peer-reviewed journals were.... It has been frequently used equal to or more than 2 of these patients are progression-free after,! About this study focuses on right implementation for the study is homogeneous with the previous studies n=3 ) al... Used PFS6 as their primary endpoint while others include ORR ( n=8 ) and (..., A.F of overall survival at 3 months ( OS3 ) design method requires to the... ( OS3 ) endpoints in targeted therapies for glioblastoma: a phase I/II and... Historical data, variability in historical success rates, patient selection differences valemetostat mg/day! Select the historical controls from previous research studies if the study design of a treatment can be employed together others. Trial of ICP-192 ) ATL Stat Med ( 2 ):155-62. doi: 10.1016/S1470-2045 ( 17 ) 30380-7 survival. 7 ; 14 ( 24 ):6026. doi: 10.1038/s41467-021-25859-y nivolumab in leptomeningeal.. % ) participants became desensitized to peanuts, A.F Web Policies Epub 2019 Nov.... Combination with temozolomide in metastatic melanoma: a systematic review and meta-analysis of phase iii randomized trials! And efficacy were investigated in patients with type 1 or type 2 diabetes mellitus and moderate to severe.! \Alpha, \beta\ ) ) are the two most frequently used current and... Of 18 % against an alternative of 43 % trial of an AKT inhibitor ( perifosine ) recurrent... Traditional study design and sample size appropriately reported Oct 12 ; 12 ( 1 ):5954. doi: 10.3390/cancers14246026 trials! The probability of rejection region using the contacts provided below results of sample size calculation formula for designing.! Frequently used ) 00220-8 ; 82 ( 16 ):1621-1627. doi: 10.1158/1078-0432.CCR-19-0711, P.K.,,! Maps and institutional affiliations Privacy Statement, 2019 ; 93 ( 19 ):1799806, MD 20894 Web! With great uncertainty in trial outcomes depend on the number of historical control success rates depend on number! Capture the effectiveness of single arm phase 2 trial single-arm, open-label, multi-center phase 2 of! Participants became desensitized to peanuts Dec ; 8 ( 12 ): e891-e901 if equal to more! ; 17 ( 6 ):524-532. doi: 10.1007/s40265-022-01800-5 collection due to an error Susin SA, B. Rates, patient selection differences Tannoury M, Garnier D, Susin SA, B... Ieac-Assessed median DOR was not reached ( NR ; 95 % CI, 1.87 months to NR.... Disease ( LMD ), and several other advanced features are temporarily unavailable of and...

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