Disclaimer, National Library of Medicine Eltarhoni K, Kamel F, Ihebunezie K, Nisar P, Soloviev M. Int J Mol Sci. Optimized image-based surrogate endpoints in targeted therapies for glioblastoma: a systematic review and meta-analysis of phase iii randomized controlled trials. Lancet Oncol. WebEVER-132-001 (NCT04454437) was a multicenter, single-arm, Phase IIb study in Chinese patients with mTNBC who failed 2 prior chemotherapy regimens. 1990;9(3):21528. 2012;11(3):2419. Neurosurg. First, if expected sample size of minimax design is close to that of optimal design, the minimax design might be a good option over the optimal design. 2020 Mar 15;39(6):698-708. doi: 10.1002/sim.8440. 2018;7(4):e1412901. Yoo, W., Kim, S., Garcia, M. et al. A complete systematic review and discussions for overcoming design issues might be important to better understand why oncology trials have shown low success rates in early phase trials. Please remove one or more studies before adding more. CNS Drugs. Please enable it to take advantage of the complete set of features! 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]. Results. We restricted the phase II clinical trials in glioblastoma to those published in 2011 or later. We also extracted the study results of study termination status after stage 1 and further investigation needed based on the efficacy. Single-arm studies have been traditionally used in Phase II oncology clinical trials. Information provided by (Responsible Party): An International, Single-Arm, Multicenter Phase 2 Trial. For instance, Silvani et al. Stat Med. 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. Please enable it to take advantage of the complete set of features! Epub 2017 Aug 16. Schmidt R, Faldum A, Kwiecien R. Adaptive designs for the one-sample log-rank test. MeSH 2013;113(1):12734. Analysis of survival data under the proportional hazards model. Two-stage designs have become popular due to large resources for implementation and comprehensible theories with various methodologic expansion in many ways for last two decades. Adaptive two-stage designs for single-arm phase IIA cancer clinical trials. Biometrics. Dowlati A, Crosby L, Remick SC, Makkar V, Levitan N. Lung Cancer. The underestimation rate of p0 was ranged from 0.1 (i.e., 10% reduction) to 0.5 (i.e., 50% reduction). 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 2003;95(19):1434-1439. Among 29 trials, only three has been completed for both stages and two studies have shown the efficacy. However, we should acknowledge there exist heterogeneities when we borrow the historical control information from other published studies due to different population conditions and quality of supportive care. Unable to load your collection due to an error, Unable to load your delegates due to an error. Estimation of clinical trial success rates and related parameters. Provided by the Springer Nature SharedIt content-sharing initiative. 2015;121(2):297302. Brenner AJ, et al. Optimal, minimax and admissible two-stage design for phase II oncology clinical trials. 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]. Rubinstein LV, et al. 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. Phase 0 clinical trials: recommendations from the task force on methodology for the development of innovative cancer therapies. The first case is to consider them co-primary endpoints and the other case two primary endpoints. 2022 Oct;23(10):1457-1476. doi: 10.1007/s11864-022-01011-w. Epub 2022 Sep 22. A total of 17 drugs were used as single therapeutic treatment with Sunitinib (SNT) and Nintedanib (NIN) from two studies each, and Temozolomide (TMZ), Bendamustine (BEN), Temsirolimus (TMS), Gimatecan (GMT), Bosutinib (BOS), Dasatinib (DAS), Tivozanib (TIV), Imipridone (IMI), Ortatzxel (ORT), Dovitinib (DOV), Perifosine (PRF), Thrombopoietin receptor (THR), and Pomalidomide (POM) from single study each. This work was supported by Ben and Catherine Ivy Foundation and Barrow Neurological Institute. The site is secure. Publisher Correction: Single-arm, open-label phase 2 trial of pembrolizumab in patients with leptomeningeal carcinomatosis. J Neurooncol. 2012;110(1):1118. WebTOCIVID-19 trial is registered in the EudraCT database with number 2020-001110-38 and in clinicaltrials.gov with ID NCT04317092. 8600 Rockville Pike The survival endpoint was introduced as Bayesian extension of Simons two-stage design and R package BayesDesign [57]. Ott PA, Elez E, Hiret S, Kim DW, Morosky A, Saraf S, Piperdi B, Mehnert JM. Would you like email updates of new search results? Phase II trial of an AKT inhibitor (perifosine) for recurrent glioblastoma. Tawbi HA, et al. Fleming TR. and transmitted securely. Designing phase 0 cancer clinical trials. Only three studies have been completed for both stages and two out of the three completed studies had shown the efficacy. This site must represent disease recurrence after completion of therapy, progressive disease on therapy, or refractory disease during induction, Myelodysplastic syndrome or any malignancy other than NB, Any systemic anti-cancer therapy within 3 weeks, Autologous stem cell transplant (ASCT) within 6 weeks prior to enrollment or ongoing toxicity due to the stem cell transplant at the discretion of the investigator, Therapeutic 131I-MIBG within 6 weeks prior to enrollment, Radiotherapy (RT) within 4 weeks prior to enrollment at any lesion site that will be identified as a target lesion to measure tumor response, Prior treatment with anti-GD2 if the patient experienced Progressive Disease (PD) while on anti-GD2 treatment, Receipt of second line chemotherapy after designation of primary refractory disease or first relapse or PD, NB in the Central Nervous System (CNS) or leptomeningeal disease within 6 months prior to enrollment, Performance status of < 50% as per the Lansky scale (patients less than 16 years of age) or Karnofsky scale (for patients aged 16 years or older), Left ventricular ejection fraction < 50% by echocardiography, Treatment with long-acting myeloid growth factor within 14 days or short-acting myeloid growth factor within 7 days prior to first dose of GM-CSF, Receipt of immunosuppressive treatment (local steroids excluded) within 4 weeks prior to enrollment, Uncontrolled seizure disorders despite anticonvulsant therapy (defined as a seizure event within 3 months prior to enrollment), Treatment with enzyme-inducing anticonvulsants including phenytoin, phenobarbital, or carbamazepine for at least 7 days prior to enrollment, Allogeneic hematopoietic stem cell transplantation (allo-SCT) or donor-lymphocyte-infusion (defined as any kind of active allogeneic lymphocyte suspension), Treatment with Hematopoietic Progenitor Cell (HPC) boost within 2 months prior to enrollment, History of allergy or known hypersensitivity to GM-CSF, yeast-derived products, or any component of GM-CSF, naxitamab, irinotecan or temozolomide, History of anaphylactic reactions CTCAE Grade 4 related to prior anti-GD2 antibody therapy, Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >5 times upper normal limit (UNL), Unacceptable kidney function at screening, defined as estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2 calculated by the 2009 revised Bedside Schwartz Equation, Significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of trial agents or to significantly increase the severity of the toxicities experienced from trial treatment, Females of childbearing potential who are pregnant, breast feeding, intend to become pregnant, or are not using adequate contraceptive methods or males who are not using adequate contraceptive methods. However, there are circumstances where the minimax designs are preferrable than the optimal design. 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. Wong CH, Siah KW, Lo AW. 2021 Oct 12;12(1):5954. doi: 10.1038/s41467-021-25859-y. 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. 2018 Sep;17(5):541-554. doi: 10.1002/pst.1878. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. J Neurooncol. We conducted a single-arm, phase 2 study of pembrolizumab in patients with solid tumor malignancies and LMD (NCT02886585). Chamberlain MC, Johnston SK. Kinders R, et al. 2019;144(2):4037. -, Grossman, S. A. et al. Intraventricular methotrexate therapy of leptomeningeal metastasis from breast carcinoma. Trials involving several arms, or randomized trials, treat randomly-selected groups of patients with different therapies in order to compare their medical outcomes. Valemetostat 200 mg orally once Accessibility 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. Findings: Between March 30, 2015, and Aug 3, 2018, 79 patients were enrolled. Kim S, Wong WK. Phase 2 single-arm trial designs typically allow Type 1 and 2 error rates up to 20% [50]. Google Scholar. Glioblastoma clinical trials: current landscape and opportunities for improvement. 2007;7(6):32534. 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. Secondary endpoints included best response in disease compartments, duration of response (DOR), pharmacokinetics, and safety. 2022 Dec 26;56:101779. doi: 10.1016/j.eclinm.2022.101779. Our simulation study showed that the proposed log-rank test performs well and is robust of misspecified weight and the sample size calculation formula also performs well. Patients pretreated with mogamulizumab had an ORR of 45.8% (4 complete and 7 partial remissions). Neyns B, et al. Altwairgi AK, et al. In this case, there are two ways to incorporate two endpoints into the trial design. The https:// ensures that you are connecting to the Epub 2022 Jan 6. 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