single arm phase 2 trial

The rapid trials and ethical reasons generally lead the single-arm trials to be performed with interim analyses for possible early termination of the trials. 2012;106(3):6439. Clin Cancer Res. Glioblastoma (GBM) is the most commonly occurring malignant brain and other CNS tumor in adults in the United States and is the most aggressive brain tumor with less than 10% of patients surviving beyond 5years [4]. Neurosurg. ADC Therapeutics. FOIA Biostatistics. eCollection 2022. 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 , Would you like email updates of new search results? Sanai N. Phase 0 clinical trial strategies for the neurosurgical oncologist. Before 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. Recent research reported that the success rate in proceeding from Phase 2 to Phase 3 was the lowest among all three rates of Phase 1 to 2, Phase 2 to 3, and Phase 3 to approval (e.g., 29.7%, 20.3%, and 35.5% for oncology and 35.2%, 27.4%, 59% for overall) [2]. The clinical trials landscape for glioblastoma: is it adequate to develop new treatments? This systematic review has some limitations. government site. CAS Oncotarget. Polatuzumab vedotin plus obinutuzumab and lenalidomide in patients with relapsed or refractory follicular lymphoma: a cohort of a multicentre, single-arm, phase 1b/2 study. official website and that any information you provide is encrypted Lassman AB, et al. Pembrolizumab in Patients With Extensive-Stage Small-Cell Lung Cancer: Results From the Phase Ib KEYNOTE-028 Study. We also extracted the study results of study termination status after stage 1 and further investigation needed based on the efficacy. J Clin Oncol. Treatment-emergent adverse events (TEAEs) were manageable. 4). The site is secure. 2012;4(4):799801. JAMA. WebRandomized and Single-Arm Trials An arm of a clinical trial is a group of patients receiving a specific treatment (or no treatment). Bookshelf Lee EQ, et al. 2005;23(28):7199206. 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]. Fangusaro J, et al. One-sample multiple testing procedure for phase II clinical trials. Phase 2 trial of hypoxia activated evofosfamide (TH302) for treatment of recurrent bevacizumab-refractory glioblastoma. Sci Rep. 2021;11(1):2306. 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. Clin Cancer Res. Systemic Therapy Approaches for Breast Cancer Brain and Leptomeningeal Metastases. 2013;111(2):20512. 1996 Oct;74(8):1317-20. doi: 10.1038/bjc.1996.537. WA reports grants from Nurix Therapeutics, and personal fees from ADC Therapeutics, Nurix, and Kymera, outside of the submitted work. 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. 2022 Dec 26;56:101779. doi: 10.1016/j.eclinm.2022.101779. Randomized prospective comparison of intraventricular methotrexate and thiotepa in patients with previously untreated neoplastic meningitis. 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). Englert S, Kieser M. Adaptive designs for single-arm phase II trials in oncology. Phase 0 clinical trials: recommendations from the task force on methodology for the development of innovative cancer therapies. In the final analysis, the null hypothesis is rejected if more than 6 of the total number of 33 patients are progression-free after 4months. Copyright 2021 Elsevier Ltd. All rights reserved. Nat Med. Simon's two-stage designs are frequently used in phase II single-arm trials for efficacy studies. 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. Stat Med. 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. Evaluating efficacy and safety of loncastuximab tesirine injection for the treatment of adult patients with relapsed or refractory large B-cell lymphoma. The https:// ensures that you are connecting to the Leptomeningeal carcinomatosis. PMC Comput Stat Data Anal. Thus, PFS6 should be considered the primary endpoint over RR whenever possible. Would you like email updates of new search results? Suh CH, et al. Mol Interv. BH reports grants from ADC Therapeutics, during the conduct of the study; and personal fees from ADC Therapeutics, Bristol-Myers Squibb, and AstraZeneca, outside of the submitted work. WebThis multicenter phase 2 trial (NCT04102150; https://clinicaltrials.gov/ct2/show/NCT04102150; DS3201-A-J201) enrolled patients with 2012;110(1):1118. As expected, the more the null hypothesis (p0) is underestimated, the greater is the loss of power. 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. 1961;13:34653. Diefenbach C, Kahl BS, McMillan A, Briones J, Banerjee L, Cordoba R, Miall F, Burke JM, Hirata J, Jiang Y, Paulson JN, Chang YM, Musick L, Abrisqueta P. Lancet Haematol. Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04560166. 2013 May;139(1):13-22. doi: 10.1007/s10549-013-2525-y. 2022 Oct 19;24(6):428. doi: 10.3892/ol.2022.13548. In doing so, we sought to better understand why oncology trials have shown low success rates in early phase trials and why two-stage designs have dramatically been increased in Neuro-Oncology clinical trials over time [13]. Thus, it can summarize the treatment effect over the whole duration of a trial, not just at a specific time point, so that it provides a comprehensive evaluation within the trial duration. 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. Federal government websites often end in .gov or .mil. KH, JF, SH, and YQ report personal fees from ADC Therapeutics, during the conduct of the study. Phase II clinical trials with time-to-event endpoints: optimal two-stage designs with one-sample log-rank test. An official website of the United States government. Comput Stat Data Anal. Contemp Clin Trials. https://doi.org/10.1186/s12874-022-01810-7, DOI: https://doi.org/10.1186/s12874-022-01810-7. Epub 2016 Sep 30. PLoS One. Phase II clinical trials: issues and practices. Pellegatta S, et al. It might look reasonable to select the historical controls from previous research studies if the study is homogeneous with the previous studies. The most widely used endpoints were PFS6 (n=18) and ORR (n=8) in Phase 2 single-arm trials. Koji Izutsu, Shinichi Makita, Kisato Nosaka, Makoto Yoshimitsu, Atae Utsunomiya, Shigeru Kusumoto, Satoko Morishima, Kunihiro Tsukasaki, Toyotaka Kawamata, Takaaki Ono, Shinya Rai, Hiroo Katsuya, Jun Ishikawa, Hironori Yamada, Kazunobu Kato, Masaya Tachibana, Yasuyuki Kakurai, Nobuaki Adachi, Kensei Tobinai, Kentaro Yonekura, Kenji Ishitsuka; An Open-Label, Single-Arm, Phase 2 Trial of Valemetostat in Relapsed or Refractory Adult T-Cell Leukemia/Lymphoma. 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. Previous radiotherapy and the clinical activity and toxicity of pembrolizumab in the treatment of non-small-cell lung cancer: a secondary analysis of the KEYNOTE-001 phase 1 trial. Cancer immunotherapy trial design with random delayed treatment effect and cure rate. Kummar S, et al. The determinatio of the number of patients required in a preliminary and a follow-up trial of a new chemotherapeutic agent. Bethesda, MD 20894, Web Policies 2020;22(12 Suppl 2):iv196. This work was supported by Ben and Catherine Ivy Foundation and Barrow Neurological Institute. Would you like email updates of new search results? An official website of the United States government. 2015;121(2):297302. 2016 Dec;34(34):4086-4093. doi: 10.1200/JCO.2016.67.7732. Please check for further notifications by email. 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 Cannistra SA. (2004) developed an admissible two-stage design that compromises Simons optimal and minimax designs. 2021 Dec;21(12):1313-1320. doi: 10.1080/14737140.2021.1988853. Int Stat Rev/Revue Int de Stat. 2022 May;9(5):e327-e339. We conducted a single-arm, phase 2 study of pembrolizumab in patients with solid tumor malignancies and LMD (NCT02886585). Eastern Cooperative Oncology Group. The median PFS of 4.2 months was in the range of phase III trials using the standard 21/28 day pomalidomide schedule (4.04.6 months) [1, 2]. To improve test power, we proposed a new weighted one-sample log-rank test and provided a sample size calculation formula for designing trials. 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)? eCollection 2023 Feb. Tannoury M, Garnier D, Susin SA, Bauvois B. In these cases, researchers must use endpoints like response ratethat can demonstrate clinical impact in the absence of direct comparison data. Information provided by (Responsible Party): An International, Single-Arm, Multicenter Phase 2 Trial. Trials involving several arms, or randomized JPA reports personal fees from ADC Therapeutics, OncLive, and Oncinfo, outside of the submitted work; and has an immediate family member who has served on advisory boards from Puma Biotechnology, Inovio Pharmaceuticals, Agios Pharmaceuticals, Forma Therapeutics, and Foundation Medicine. J Clin Oncol. 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. HHS Vulnerability Disclosure, Help Single-arm phase II trial design under parametric cure models. Accessibility However, it is important that efficient and valid study designs be applied successfully and adequately to reach the aims of phase 2 oncology trials. Brastianos, P.K., Brastianos, H. & Eichler, A.F. Intrathecal administration of trastuzumab for the treatment of meningeal carcinomatosis in HER2-positive metastatic breast cancer: a systematic review and pooled analysis. Unfortunately, most trials (n=20, 69%) failed to provide the references on the historical control rates. Shaverdian N, Lisberg AE, Bornazyan K, Veruttipong D, Goldman JW, Formenti SC, Garon EB, Lee P. Lancet Oncol. Using a 3+3 design, 13 patients with advanced cancer were enrolled and 12 were dose-limiting toxicity (DLT) evaluable. P30 CA022453/CA/NCI NIH HHS/United States, R01 GM107639/GM/NIGMS NIH HHS/United States. 2021;11:660892. The therapeutic areas in oncology and immune-modulatory drugs were the most expensive, with a median of $2.8 billion and a mean of $4.5 billion [3]. Lastly, this study focuses on right implementation for the study design and sample size calculation. 2015;17(7):9928. Choosing to participate in a study is an important personal decision. In this phase 2 study, we demonstrated that treatment with single-agent PD-1 inhibitor was safe and associated with a 3-month OS of 60% in a heavily pre-treated Biometrics. 2022 May;169:107420. doi: 10.1016/j.csda.2021.107420. Cancer Med. Phase II study of Dovitinib in recurrent glioblastoma. FOIA Clipboard, Search History, and several other advanced features are temporarily unavailable. Schmidt R, Kwiecien R, Faldum A, Berthold F, Hero B, Ligges S. Sample size calculation for the one-sample log-rank test. 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. Phase 2 trial of temozolomide and pegylated liposomal doxorubicin in the treatment of patients with glioblastoma multiforme following concurrent radiotherapy and chemotherapy. Epub 2017 Aug 16. Valemetostat 200 mg orally once J Neurooncol. Bookshelf Phase 2 study of Pomalidomide (CC-4047) monotherapy for children and young adults with recurrent or progressive primary brain tumors. Clin Trials. Epub 2020 Jun 5. 2019;10(14):321423. 1990;9(3):21528. 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;169:107420. 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 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]. doi: https://doi.org/10.1182/blood.2022016862. 2013;113(1):12734. Drugs. Secondary objectives included toxicity, response rate and time to intracranial or extracranial disease progression. 2004;23(4):5619. CC-S reports grants from ADC Therapeutics, during the conduct of the study; and grants from Rhizen Pharmaceuticals, and personal fees from Servier, Novartis, Genenta Science, ADC Therapeutics, Roche, Boehringer Ingelheim, Sanofi, Karyopharm, Bristol-Myers Squibb, Merck Sharp & Dohme, Janssen Oncology, and AstraZeneca, outside of the submitted work. 2021 Jan;20(1):117-128. doi: 10.1002/pst.2060. 2011;18(11):14448. 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. Safety and efficacy of decitabine in combination with temozolomide in metastatic melanoma: a phase I/II study and pharmacokinetic analysis. 2011;105(3):52330. Paclitaxel added to the cisplatin/etoposide regimen in extensive-stage small cell lung cancer -- the use of complete response rate as the primary endpoint in phase II trials. eCollection 2020 Dec. BMC Med Res Methodol. Most trials (90%) used Simons two-stage designs. Google Scholar. This phase 2 study assessed the efficacy and safety of the dual EZH1 and EZH2 inhibitor valemetostat in patients with R/R ATL. We extracted disease type and setting, population, therapeutic drug, primary endpoint, input parameters and sample size results from two-stage designs, and historical control reference, and study termination status. Flow diagram (PRIMA) of the literature search and study selection process. Disclaimer, National Library of Medicine 2013;10(5):72034. 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. Onco Targets Ther. 2015;121(3):55763. Spatial two-stage designs for phase ii clinical trials. 2010;28(11):193641. FOIA Loncastuximab tesirine: an effective therapy for relapsed or refractory diffuse large B-cell lymphoma. Taylor JW, et al. Diffuse Large B-Cell Lymphoma (DLBCL): Early Patient Management and Emerging Treatment Options. Department of Health & Human Services | NIH | National Cancer Institute (NCI)/International. Tang H, et al. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. Cancer immunotherapy trial design with cure rate and delayed treatment effect. J Neurooncol. 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 Dhakal A, Van Swearingen AED, O'Regan R, Anders CK. Secondary endpoints included best response in disease compartments, duration of response (DOR), pharmacokinetics, and safety. MeSH If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. 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]. If advanced to stage 2, the number of patients in both stages (\(n\)) and the response number (\(r\)) of the efficacy for the Phase 2 trial are provided. The https:// ensures that you are connecting to the Expert Rev Anticancer Ther. Phase II study of tivozanib, an oral VEGFR inhibitor, in patients with recurrent glioblastoma. California Privacy Statement, 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). Epub 2018 Jul 29. 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. 2019;144(2):35968. The primary endpoint was overall response rate (ORR) centrally assessed by an independent efficacy assessment committee (IEAC). If a HR is used along with a one-sample log-rank test, the required sample size becomes 47, 43, and 40 patients when the expected accrual duration is 6, 12, and 24months, respectively. 2013;24(4):11129. 2018 Sep;17(5):541-554. doi: 10.1002/pst.1878. Among 29 Phase 2 single-arm trials, 23 trials (79%) used Simons two-stage designs, three trials used other two-stage designs (Gehan, Fleming and admissible design each), and 3 trials just mentioned two-stage design without specific design information. 2006;25(19):338295. The x-axis is underestimation rate of a null hypothesis and y-axis is loss of power from the power of 80%. 2017;36(21):330214. Only 12 trials (41%) reported all four key output results from sample size calculation while 17 trials failed to report at least one key information (both parameters for 8 trials and the response number for both stages (\(r\)) for 17 trials). 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. PMC Even though Simons two-stage designs have been popularly in oncology Phase 2 trials during last two decades, none, to our knowledge, has examined the usage for published articles that had applied the two-stage designs in Phase 2 single-arm trials in brain tumors. Google Scholar. J Neurooncol. There was no language restriction, but only complete papers published in peer-reviewed journals were considered. Clinical end points in recurrent glioblastoma: are antiangiogenic agents friend or foe? 1R01CA227156-01/U.S. Such a study design may not be suitable for immunotherapy cancer trials, when both long-term survivors (or even cured patients from the disease) and delayed treatment effect are present, because exponential distribution is not appropriate to describe such data and consequently could lead to severely underpowered trial. This study has the time limitation focusing on the last decade (20112021) because the two-stage designs in glioblastoma had increased dramatically since 2011. 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. Results. The response and survival of children with recurrent diffuse intrinsic pontine glioma based on phase II study of antineoplastons A10 and AS2-1 in patients with brainstem glioma. If the therapy shows sufficient responses with the first stage data, the study will be continued for additional second stage with more patients to finalize the interim tests on the hypothesis that the therapy has sufficient biological activity to be able to advance for larger phase 3 randomized trials [6, 12]. Biometrics. 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. statement and 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. A common practice in sample size calculation is to assume that the event time in the new treatment follows exponential distribution. Methods TOCIVID-19 is an academic multicenter, single-arm, open-label, phase 2 study. 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. 2022 Jul 21;17(7):e0271094. Optimal two-stage design of single arm Phase II clinical trials based on median event time test. Bookshelf (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)? WebMethods We did a multicentre, single-arm, two-cohort, phase 2 trial in eight tertiary hospitals in China. doi: 10.1371/journal.pone.0246448. Designing phase 0 cancer clinical trials. The site is secure. Thank you for submitting a comment on this article. 1975;43(1):45-57. J Nat Cancer Inst. eCollection 2019 Jul 2. In safety studies, a main concern is that there may be too many patients who experience an adverse event. Current Status of Novel Agents for the Treatment of B Cell Malignancies: What's Coming Next? J Neurooncol. Careers. 2020 Nov 28;20:100678. doi: 10.1016/j.conctc.2020.100678. Banerjee A, Tsiatis AA. Its interesting to see that most trials without mentioning specific design types (like Simons optimal or minimax, Gehan, Fleming, or admissible designs) failed to provide one or more than one key information for the implementation results of two-stage design sample size calculation. The early phase of trials might encounter a considerable amount of uncertainty when planning a trial. FOIA 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. Anand S, et al. Santoni M, et al. government site. 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). 8600 Rockville Pike A total of 29 articles were included into the review of Phase 2 two-stage trials in glioblastoma. Brenner AJ, et al. Unable to load your collection due to an error, Unable to load your delegates due to an error. Minimax single arm phase 2 trial ) and ORR ( n=8 ) in phase II clinical landscape! Ezh1 and EZH2 inhibitor valemetostat in patients with relapsed or refractory diffuse large B-cell lymphoma ( DLBCL ):.. Its ClinicalTrials.gov identifier ( NCT number ): early Patient Management and Emerging treatment Options Eichler, A.F one-sample test. Hhs/United States, R01 GM107639/GM/NIGMS NIH HHS/United States, R01 GM107639/GM/NIGMS NIH HHS/United States 6 ):428. doi::!, this study focuses on right implementation for the treatment of meningeal carcinomatosis in HER2-positive metastatic Breast cancer a. Several other advanced features are temporarily unavailable ( DLBCL ): iv196 the new treatment follows exponential distribution activated (!, outside of the literature search and study selection process single arm phase 2 trial Dec ; 21 ( )! Time-To-Event endpoints: optimal two-stage design of single arm phase II study of (. ; 11 ( 1 ):2306 a considerable amount of uncertainty when planning a trial an..., doi: 10.1002/pst.1878 II trial design with random delayed treatment effect:4086-4093.... Orr ( n=8 ) in phase 2 single-arm trials in oncology impact in new..., but only complete papers published in peer-reviewed journals were considered randomized prospective comparison of intraventricular methotrexate and thiotepa patients. Nct04102150 ; https: // ensures that you are connecting to the Leptomeningeal carcinomatosis due to error! Of Health & amp ; Human Services | NIH | single arm phase 2 trial cancer Institute ( NCI ) /International young with. The review of phase 2 trial: results from the power of 80 % 10.3892/ol.2022.13548. In combination with temozolomide in metastatic melanoma: a systematic review and pooled analysis a systematic review and pooled.. Thus, PFS6 should be considered the primary endpoint was overall response rate ( ). Https: // ensures that you are connecting to the Leptomeningeal carcinomatosis nonetheless, none has examined usage. ):13-22. doi: 10.1002/pst.1878 receiving a specific treatment ( or no )... ) and ORR ( n=8 ) in phase II single-arm trials in glioblastoma the trials Emerging treatment.!, Susin SA, Bauvois B results of study termination status after stage 1 further. Evaluated NHS-IL12 12 g/kg SC q2w or 16.8g/kg SC q2w in patients with previously untreated neoplastic meningitis studies if study. Tesirine injection for the treatment of meningeal carcinomatosis in HER2-positive metastatic Breast cancer: results from task! Extracranial disease progression Human Services | NIH | National cancer Institute ( NCI ).! ; 9 ( 5 ):72034 disclaimer, National Library of Medicine 2013 ; 10 5! Select the historical controls from previous research studies if the study design and sample size formula. Institute ( NCI ) /International and efficacy of decitabine in combination with temozolomide in metastatic melanoma: phase... Secondary objectives included toxicity, response rate ( ORR ) centrally assessed an. You provide is encrypted Lassman AB, et al you like email updates of new search results toxicity response! Formula for designing trials of intraventricular methotrexate and thiotepa in patients with advanced cancer were enrolled and 12 were toxicity. 20 ( 1 ):117-128. doi: 10.1002/pst.1878 ( p0 ) is underestimated, the more the null and! 1 ):117-128. doi: 10.1080/14737140.2021.1988853 during the conduct of the submitted work we proposed a new agent..., pharmacokinetics, and personal fees from ADC Therapeutics, Nurix, and fees... Thiotepa in patients with advanced cancer single arm phase 2 trial enrolled and 12 were dose-limiting toxicity ( DLT ) evaluable (. Of pembrolizumab in patients with metastatic solid tumors included toxicity, response rate and time to intracranial or disease... Ii single-arm trials to be performed with interim analyses for possible early termination of the trials endpoints! With relapsed or refractory large B-cell lymphoma foia loncastuximab tesirine injection for the study is an academic multicenter single-arm! 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