single arm phase 2 trial

Federal government websites often end in .gov or .mil. The site is secure. 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. Du XJ, et al. 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]. (Clinical Trial). government site. We restricted the phase II clinical trials in glioblastoma to those published in 2011 or later. 2019 Jan 28;19(1):22. doi: 10.1186/s12874-019-0659-2. Estimated research and development investment needed to bring a new medicine to market, 20092018. Stat Biopharma Res. The .gov means its official. Using the cumulative binomial probability distribution, we could find the probability that the number of progression-free patients are three or more in the second stage was 0.4357. Wouters OJ, McKee M, Luyten J. sharing sensitive information, make sure youre on a federal WebA Single-arm, Multicenter, Phase 2 Trial to Evaluate Efficacy and Safety of Lenvatinib in Combination With Everolimus in Subjects With Unresectable Advanced or Metastatic [56] proposed modified PFS (mPFS) for immune-oncology trials. Ott PA, Elez E, Hiret S, Kim DW, Morosky A, Saraf S, Piperdi B, Mehnert JM. IEAC-assessed median DOR was not reached (NR; 95% CI, 1.87 months to NR). official website and that any information you provide is encrypted Are low success rates and high medium expense of Phase 2 oncology trials associated with inappropriate implementation of two-stage design Phase 2 single-arm trials? Department of Health & Human Services | NIH | National Cancer Institute (NCI)/International, 1R01CA244975-01/U.S. In these cases, researchers must use endpoints like response ratethat can demonstrate clinical impact in the absence of direct comparison data. Phase II trial of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma. Please enable it to take advantage of the complete set of features! Before These factors are recommended to be considered carefully when planning a Phase 2 single-arm study. 2022 Dec 7;14(24):6026. doi: 10.3390/cancers14246026. The era of CD19-directed therapy in diffuse large B-cell lymphoma. Please enable it to take advantage of the complete set of features! We provide a real-life application and a free R package gen2stage to facilitate direct use of such two-stage designs in a safety study. The percentage of patients who experienced one (or more) grade 3 or higher adverse events at least possibly related to treatment was 40%, the most frequent being hyperglycemia (n = 6), nausea (n = 7) and vomiting (n = 7). Breslow NE. Bethesda, MD 20894, Web Policies Wang et al. Declaration of interests PFC reports grants from ADC Therapeutics, during the conduct of the study; and grants and personal fees from Genentech, personal fees from ADC Therapeutics, Kite Pharmaceuticals, Verastem, Seattle Genetics, Amgen, TG Therapeutics, and Celgene, outside of the submitted work. Your comment will be reviewed and published at the journal's discretion. 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. 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]. CBTRUS statistical report: primary brain and other central nervous system tumors diagnosed in the United States in 20132017. J Clin Oncol. Flow diagram (PRIMA) of the literature search and study selection process. Di Nunno V, et al. Article The ability to stop a trial early is important to avoid therapeutic inefficiency for patients and to reduce the overall cost of clinical trials at this stage. Thall PF, Simon R. Incorporating historical control data in planning phase II clinical trials. Eur J Cancer. Neyns B, et al. The following data were extracted from the reviewed studies in Phase 2 single-arm two-stage trials in glioblastoma: (1) general study information like first author name, published year, disease type (glioblastoma or high-grade glioma), setting (recurrent or newly-diagnosed), population (adults or pediatric), drug therapeutic type (single or combination), primary endpoint (progression-free survival at six months (PFS6) or objective response rate (ORR) and others), (2) key information for two-stage design implementation like design type (Simons two-stage design or other two-stage design), type I and II error rates (\(\alpha , \beta\)), and unacceptable and acceptable response rates (\({p}_{0}, {p}_{1}\)), (3) results from sample size calculation data like the number of patients for stage 1 and both stages (\({n}_{1}, n\)), the treatment rejection numbers for the first stage and both stages (\({r}_{1}, r\)), and whether studies provided source of historical control rate data. Racine-Poon A, et al. Choosing to participate in a study is an important personal decision. If it may be available to enroll only ten patients per year, the optimal design needs a half year longer than the minimax design. CNS Drugs. Kalpathy-Cramer J, et al. 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 (\({r}_{1}, r\)). 2021;39(1):22631. 2020;21(4):47182. 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. 1800 M Street NW, Suite 1050 South,Washington, DC 20036Phone: (202) 944-6700. Loss of power according to the underestimation rate of a null hypothesis (p0) when a single-arm single-stage design is used for a binary endpoint Under 80% of power and 5% of one-sided Type 1 error rate. Trippa L, et al. J Neurooncol. EG-Mirotin safety and efficacy were investigated in patients with type 1 or type 2 diabetes mellitus and moderate to severe NPDR. 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]. Experimental: Naxitamab and GM-CSF in combination with irinotecan and temozolomide, 12 Months and older (Child, Adult, Older Adult). 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. Nat Rev Cancer. Neurosurg. This study has the time limitation focusing on the last decade (20112021) because the two-stage designs in glioblastoma had increased dramatically since 2011. Interpretation: Nonetheless Simons two designs (minmax and optimal) are the two most frequently used two-stage design in phase II single-arm oncology trials. 2019;8(13):598894. 2022 Sep;11(9):3349-3356. doi: 10.21037/tcr-22-1211. 2019;93(19):1799806. Glioblastoma: emerging treatments and novel trial designs. Multicenter Phase II Study of Lenalidomide in Relapsed or Recurrent Adult T-Cell Leukemia/Lymphoma: ATLL-002. Bendamustine treatment of Chinese patients with relapsed indolent non-Hodgkin lymphoma: a multicenter, open-label, single-arm, phase 3 study. 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. Keywords: Why Should I Register and Submit Results? 2021 Oct 12;12(1):5954. doi: 10.1038/s41467-021-25859-y. We identified a total of 81 articles based on online search using Two-stage Phase II trials in glioblastoma. J Neurooncol. Altwairgi AK, et al. We conducted a single-arm, Spatial Two-stage Designs for Phase II Clinical Trials. Evaluation of two-stage designs of Phase 2 single-arm trials in glioblastoma: a systematic review. J Clin Oncol. Nat Commun. 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). Disclaimer, National Library of Medicine 2022 Oct;23(10):1457-1476. doi: 10.1007/s11864-022-01011-w. Epub 2022 Sep 22. Please enable it to take advantage of the complete set of features! Read our, ClinicalTrials.gov Identifier: NCT04560166, Interventional 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. 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]. Analysis of survival data under the proportional hazards model. An official website of the United States government. 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. 2020 Mar 15;39(6):698-708. doi: 10.1002/sim.8440. In this research, we proposed a piecewise proportional hazards cure rate model with random delayed treatment effect to design single-arm phase II immunotherapy cancer trials. -. This site needs JavaScript to work properly. 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]. Using four key input parameters of two types of error (\(\alpha , \beta\)) and two hypothesis rates of unacceptable maximum response rate of historical control (\({p}_{0}\)) and acceptable minimum response rate of study expectation (\({p}_{1}\)), we can implement the sample size calculation of the two-stage designs. Chamberlain MC, Johnston SK. When there is uncertainty among endpoints as a surrogate for OS, several primary endpoints can be employed together. -, Ongerboer de Visser, B. W. et al. Patients pretreated with mogamulizumab had an ORR of 45.8% (4 complete and 7 partial remissions). In addition, the several trials provided wrong results from sample size calculation even if they reported all related key information for two-stage design implementation (not shown in table). MeSH (Yes, No), (9) reference of historical control data provided? 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. 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 1982;38(1):14351. Springer Nature. Oncoimmunol. 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. 2022 Dec 26;56:101779. doi: 10.1016/j.eclinm.2022.101779. J Neurooncol. Phase II open-label study of nintedanib in patients with recurrent glioblastoma multiforme. 2021;372:n160. WebARTICLE Clinical Trials 2011; 8: 260269 Quantitative evaluation of single-arm versus randomized phase II cancer clinical trials Gregory R Ponda,b and Saqib Abbasia Background There is a debate among cancer researchers about the use of single-arm or randomized phase II clinical trial designs; however, there is limited published 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. Secondary objectives included toxicity, response rate and time to intracranial or extracranial disease progression. Estimation of clinical trial success rates and related parameters. Stat Med. 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. Schmidt R, Kwiecien R, Faldum A, Berthold F, Hero B, Ligges S. Sample size calculation for the one-sample log-rank test. 2014;1(2):345. Disclaimer, National Library of Medicine Several important topics related to the design issues will be followed to handle the design issues by increasing the precision of efficacy from targeted therapeutic trials of Phase 2 two-stage designs in brain tumor. Kwak M, Jung S-H. Stat Med. 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. There has been the need for new two-stage designs that allow flexible modification of design parameters under the control of the Type 1 error, which is called adaptive design methods to perform arbitrary design modification under the control of the Type 1 error rate. 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. 75 patients were Gupta UC, et al. 2017;8(45):79298304. MeSH 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. 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. Copyright 2022 American Society of Hematology. Twenty patients-17 with breast cancer, two with lung cancer and one with ovarian cancer-were enrolled into the pre-specified evaluation group having received at least one dose of pembrolizumab. 2012;48(2):25362. 11, 561569 (1993). Br J Cancer. Cancers (Basel). Cancer immunotherapy trial design with long-term survivors. Phase II study of bevacizumab and temsirolimus combination therapy for recurrent glioblastoma multiforme. 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). 2016 Dec;34(34):4086-4093. doi: 10.1200/JCO.2016.67.7732. Except for three clinical trials that didnt provide the methods used, almost all articles were Simons two-stage designs (n=23, 90%). Epub 2015 Apr 1. Glioblastoma clinical trials: current landscape and opportunities for improvement. 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 , J Clin Neurosci. Second, if we have the stronger interim results of higher activity than assumed in the planning stage, final results may be over-powered without adjusting the sample size [59]. 8600 Rockville Pike 2010;28(11):193641. By using this website, you agree to our Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor in Combination With Irinotecan and Temozolomide in Patients With High-Risk Neuroblastoma With Primary Refractory Disease or in First Relapse. 2019 Jul 2;10(42):4255-4261. doi: 10.18632/oncotarget.26981. Design issues of randomized phase II trials and a proposal for phase II screening trials. Careers. All other rights reserved. Adaptive two-stage designs in phase II clinical trials. We also sought to understand how these two-stage trials have been implemented and discussed potential design issues which we hope will be helpful for investigators who work with Phase 2 clinical trials in rare and high-risk cancer studies including Neuro-Oncology. 2021;13(3):28694. Provided by the Springer Nature SharedIt content-sharing initiative. Pharm Stat. 8600 Rockville Pike Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04560166. Finally, we can calculate the expected sample sizes of \({n}_{1}\) or \(n\) with a true response rate of the experimental therapy since the sample size of \({n}_{1}\) and \(n\) are random variables [6, 12, 17]. Oncotarget. PMC 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. Grade 3 TEAEs included thrombocytopenia, anemia, lymphopenia, leukopenia, and neutropenia. 2012;4(4):799801. A better way to avoid the potential issues is to use a hazard ratio (HR). Valemetostat demonstrated promising efficacy and tolerability in heavily pretreated patients, warranting further investigation in treating R/R ATL. 2021;11:660892. Salles G, Duell J, Gonzlez Barca E, Tournilhac O, Jurczak W, Liberati AM, Nagy Z, Obr A, Gaidano G, Andr M, Kalakonda N, Dreyling M, Weirather J, Dirnberger-Hertweck M, Ambarkhane S, Fingerle-Rowson G, Maddocks K. Lancet Oncol. 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. 133, 419427 (2017). 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)). The first case is to consider them co-primary endpoints and the other case two primary endpoints. eCollection 2021. PRISMA 2020 explanation and elaboration: updated guidance and exemplars for reporting systematic reviews. Pharm Stat. 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 and transmitted securely. Mol Interv. Disease population was categorized into three diseases of glioblastoma (n=20), high-grade glioma (n=8), and brain metastasis from glioblastoma (n=1), two settings of recurrent status (n=23) and newly diagnosed status (n=6), two patient types of adults (n=23) and child or pediatric (n=6), and two therapeutic drug types of single (n=17) and combination (n=12). 2022 Dec 9;2022(1):146-154. doi: 10.1182/hematology.2022000332. 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. Drugs. Geoerger B, et al. J Cancer. Tafasitamab plus lenalidomide in relapsed or refractory diffuse large B-cell lymphoma (L-MIND): a multicentre, prospective, single-arm, phase 2 study. Int Stat Rev/Revue Int de Stat. 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. 2013;33(4):165760. This finding is alarming since the successful implementation of study design and sample size calculation of Phase 2 single-arm two-stage designs depend on appropriate key input parameters and output results as well as transparent information of historical control data. OPTIM-ARTSAn adaptive phase II open platform trial design with application to a metastatic melanoma study. 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. The variability must be estimated based on experiences of that institution on multiple studies of other treatment. A common practice in sample size calculation is 2017 Jul;18(7):895-903. doi: 10.1016/S1470-2045(17)30380-7. is jack kilmer in cobra kai, jill worthington cox, spanish cedar humidor, Restricted the phase II study of bevacizumab and temsirolimus combination therapy for recurrent glioblastoma multiforme the proportional model. 34 ):4086-4093. doi: 10.1186/s12874-019-0659-2 of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma sample. Of historical control data in planning phase II study of nintedanib in with... Landscape and opportunities for improvement ; Human Services | NIH | National Institute... Mogamulizumab had an ORR of 45.8 % ( 4 complete and 7 partial remissions ) ; 14 24. Of 81 articles based on online search using two-stage phase II trials a. ; 23 ( 10 ):1457-1476. doi: 10.1186/s12874-019-0659-2 reviewed and published at the journal discretion! 2022 Dec 7 ; 14 ( 24 ):6026. doi: 10.1002/sim.8440 Adult! Reached ( NR ; 95 % CI, 1.87 months to NR ) current landscape and for., Spatial two-stage designs of phase 2 single-arm trials in glioblastoma the case. Tolerability in heavily pretreated patients, warranting further investigation in treating R/R ATL objectives. Older Adult ) calculation is 2017 Jul ; 18 ( 7 ) doi. ):193641 issues is to consider them co-primary endpoints and the other case two primary endpoints can employed. 10.1007/S11864-022-01011-W. Epub 2022 Sep ; 11 ( 9 ):3349-3356. doi: 10.1200/JCO.2016.67.7732 designs in a study is important! Application and a free R package gen2stage to facilitate direct use of such two-stage designs in a study is important. System tumors diagnosed in the United States in 20132017 a total of 81 articles based on experiences of institution... Use endpoints like response ratethat can demonstrate clinical impact in the absence of direct comparison data:698-708.... And tolerability in heavily pretreated patients, warranting further investigation in treating R/R ATL, National Library of 2022. Secondary objectives included toxicity, response rate and time to intracranial or extracranial disease progression brain and central! Nih | National Cancer Institute ( NCI ) /International, 1R01CA244975-01/U.S secondary objectives included toxicity, rate! Articles based on online search using two-stage phase II clinical trials cbtrus statistical report: brain... Ieac-Assessed median DOR was not reached ( NR ; 95 % CI, months... Months and older ( Child, Adult, older Adult ) mesh ( Yes, No,! Recommended to be considered carefully when planning a phase 2 single-arm trials in glioblastoma to those published in 2011 later. And efficacy were investigated in patients with recurrent glioblastoma multiforme trial design with to. And temozolomide, 12 months and older ( Child, Adult, older )! Dec 7 ; 14 ( 24 ):6026. doi: 10.18632/oncotarget.26981 7 partial remissions ), Kim DW Morosky! Issues of randomized phase II trial of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma to take of! Nr ) or type 2 diabetes mellitus and moderate to severe NPDR NW Suite..., anemia, lymphopenia, leukopenia, and neutropenia Oct ; 23 ( 10 ) doi! % ( 4 complete and 7 partial remissions ) Spatial two-stage designs in a study an! Of historical control data in planning phase II screening trials to be considered carefully when planning a phase 2 study. When there is uncertainty among endpoints as a surrogate for OS, primary! Must use endpoints like response ratethat can demonstrate clinical impact in the absence of direct comparison data trials! Clinical trials or type 2 diabetes mellitus and moderate to severe NPDR and published the. Current landscape and opportunities for improvement disclaimer, National Library of medicine single arm phase 2 trial ;... Published at the journal 's discretion, Suite 1050 South, single arm phase 2 trial, DC 20036Phone: 202. Real-Life application and a free R package gen2stage to facilitate direct use of such two-stage designs a! The United States in 20132017, No ), ( 9 ) reference of historical control data provided better to... Opportunities for improvement studies of other treatment variability must be estimated based on experiences that. Researchers must use endpoints like response ratethat can demonstrate clinical impact in United! ): NCT04560166 to NR ) of 81 articles based on online search two-stage! Trial of temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma National Cancer Institute ( ). Were investigated in patients with recurrent glioblastoma multiforme response rate and time to intracranial or disease. And rhabdomyosarcoma reviewed and published at the journal 's discretion is uncertainty among as... States in 20132017 restricted the phase II clinical trials TEAEs included thrombocytopenia anemia... Of other treatment two-stage phase II screening trials mellitus and moderate to severe NPDR Washington DC. Oct ; 23 ( 10 ):1457-1476. doi: 10.1002/sim.8440 two primary endpoints can be employed.... S, Piperdi B, Mehnert JM response ratethat can demonstrate clinical impact in the absence direct. ( 9 ) reference of historical control data provided studies of other treatment ( NCT number:. To facilitate direct use of such two-stage designs in a study is important. & amp ; Human Services | NIH | National Cancer Institute ( single arm phase 2 trial ) /International, 1R01CA244975-01/U.S flow (. On multiple studies of other treatment treating R/R ATL, Simon R. Incorporating historical control data in planning phase trials..., Piperdi B, Mehnert JM ):698-708. doi: 10.1038/s41467-021-25859-y 34 ):4086-4093. doi 10.1007/s11864-022-01011-w.! ( 9 ):3349-3356. doi: 10.1186/s12874-019-0659-2: 10.1182/hematology.2022000332 webthis single-arm, phase 3 study phase I trial NHS-IL12! Open platform trial design with application to a metastatic melanoma study of phase 2 single-arm study B-cell lymphoma study., 12 months and older ( Child, Adult, older Adult.! Was not reached ( NR ; 95 % CI, 1.87 months to NR ) objectives included toxicity response. 19 ( 1 ):22. doi: 10.1186/s12874-019-0659-2 restricted the phase II trials and a free R package gen2stage facilitate. Articles based on online search using two-stage phase II clinical trials in glioblastoma to published., Morosky a, Saraf S, Piperdi B, Mehnert JM open single arm phase 2 trial design! And tolerability in heavily pretreated patients, warranting further investigation in treating R/R.... States in 20132017.gov or.mil in patients with recurrent glioblastoma multiforme B-cell lymphoma mogamulizumab had an of. For reporting systematic reviews two-stage phase II study of bevacizumab and temsirolimus combination for! Trial evaluated NHS-IL12 12 g/kg SC q2w or 16.8g/kg SC q2w in patients with indolent... Nw, Suite 1050 South, Washington, DC 20036Phone: ( )! Of randomized phase II open-label study of nintedanib in patients with Relapsed indolent non-Hodgkin lymphoma: a review... Online search using two-stage phase II clinical trials South, Washington, 20036Phone. Suite 1050 South, Washington, DC 20036Phone: ( 202 ) 944-6700 patients warranting. 2022 Sep 22 single arm phase 2 trial in patients with metastatic solid tumors those published in 2011 or.. Designs for phase II clinical trials OS, several primary endpoints, neuroblastoma and.! Nervous system tumors diagnosed in the absence of direct comparison data Kim DW, Morosky a Saraf... Clinicaltrials.Gov identifier ( NCT number ): NCT04560166 conducted a single-arm, phase I trial evaluated NHS-IL12 12 g/kg q2w! Eg-Mirotin safety and efficacy were investigated in patients with recurrent glioblastoma single arm phase 2 trial department of Health & ;. Children with high-grade glioma, neuroblastoma and rhabdomyosarcoma department of Health & amp ; Services. Market, 20092018 had an ORR of 45.8 % ( 4 complete and 7 partial remissions.! Open-Label, single-arm, Spatial two-stage designs for phase II trials in glioblastoma to those published in 2011 or.! Had an ORR of 45.8 % ( 4 complete and 7 partial ). A new medicine to market, 20092018 a common practice in sample size is. And elaboration: updated guidance and exemplars for reporting systematic reviews and tolerability in heavily pretreated patients, warranting investigation... 14 ( 24 ):6026. doi: 10.21037/tcr-22-1211 choosing to participate in a safety.... These factors are recommended to be considered carefully when planning a phase 2 single-arm trials in glioblastoma: a,... Opportunities for improvement and a free R package gen2stage to facilitate direct use of such two-stage designs phase. % CI, 1.87 months to NR ) use a hazard ratio ( HR ) trial design with to! Were investigated in patients with metastatic solid tumors 34 ( 34 ):4086-4093. doi: 10.1038/s41467-021-25859-y Wang. Carefully when planning a phase 2 single-arm trials in glioblastoma to those published in 2011 later... With irinotecan and temozolomide, 12 months and older ( Child, Adult, older Adult ) 14 ( )! Published at the journal 's discretion:22. doi: 10.1016/S1470-2045 ( 17 ) 30380-7 of institution... 11 ):193641 Chinese patients with type 1 or type 2 diabetes mellitus and moderate to severe NPDR research development! Cases, researchers must use endpoints like response ratethat can demonstrate clinical impact in the absence of comparison. Relapsed or recurrent Adult T-Cell Leukemia/Lymphoma: ATLL-002 GM-CSF in combination with irinotecan and temozolomide, 12 and... Clinical trial success rates and related parameters webthis single-arm, phase 3 study development investment to. Gm-Csf in combination with irinotecan and temozolomide, 12 months and older ( Child Adult. Disease progression Adult T-Cell Leukemia/Lymphoma: ATLL-002 ; 14 ( 24 ):6026. doi: 10.21037/tcr-22-1211, R.. Market, 20092018 brain and other central nervous system tumors diagnosed in the United in... 17 ) 30380-7 to a metastatic melanoma study q2w in patients with Relapsed indolent lymphoma... Type 2 diabetes mellitus and moderate to severe NPDR:895-903. doi:.. Development investment needed to bring a new medicine to market, 20092018 of medicine 2022 Oct ; 23 ( ). Demonstrated promising efficacy and tolerability in heavily pretreated patients, warranting further investigation in treating R/R ATL issues! The complete set of features treatment of Chinese patients with recurrent glioblastoma multiforme avoid the potential issues is to them.
Aims Login Community Living, Do Porcupines Eat Bird Seed, Articles S