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Journal articles on the topic 'Single-arm trial'

Author: Grafiati

Published: 3 June 2025

Last updated: 31 July 2025

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1

Lasch, Florian, Kristina Weber, Mwe Mwe Chao, and Armin Koch. "A plea to provide best evidence in trials under sample-size restrictions: the example of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients." Orphanet Journal of Rare Diseases 12, no. 1 (2017): 102. https://doi.org/10.1186/s13023-017-0655-8.

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In planning a clinical trial for demonstrating the efficacy of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients we had to discuss the need for a randomized controlled trial particularly under sample-size restrictions as very promising results were available from a single-arm clinical trial. Unfortunately, at a later stage, we had to suffer from the fact that single-arm clinical trials may sometimes mislead. When revisiting our planning at a later stage of a grant application, results of a randomized controlled trial had become available which were less impressiv

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Zou, Denise, Eileen Zhang, Sherry Wu, and Virgil Rose. "Use of Single-Arm Trials in FDA Approvals of Treatments in Relapsed or Refractory B-Cell Lymphoma." Blood 142, Supplement 1 (2023): 7250. http://dx.doi.org/10.1182/blood-2023-189855.

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Background: Single-arm trials are commonly used to evaluate therapies targeting life-threatening or rare diseases (e.g., late-stage cancers that are relapsed or refractory to prior interventions). A number of oncology products supported by single-arm trials have been approved by the United States Food and Drug Administration (FDA). The draft guidance issued by the FDA on 24 March 2023 recommended key considerations for trial design and analysis when a single-arm trial is considered appropriate to support accelerated approval. The objective of this study was to assess how often anticancer drugs

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Estey, Elihu H., and Peter F. Thall. "New designs for phase 2 clinical trials." Blood 102, no. 2 (2003): 442–48. http://dx.doi.org/10.1182/blood-2002-09-2937.

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AbstractConventional phase 2 clinical trials are typically single-arm experiments, with outcome characterized by one binary “response” variable. Clinical investigators are poorly served by such conventional methodology. We contend that phase 2 trials are inherently comparative, with the results of the comparison determining whether to conduct a subsequent phase 3 trial. When different treatments are studied in separate single-arm trials, actual differences between response rates associated with the treatments, “treatment effects,” are confounded with differences between the trials, “trial effe

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Grayling, Michael J., Munyaradzi Dimairo, Adrian P. Mander, and Thomas F. Jaki. "A Review of Perspectives on the Use of Randomization in Phase II Oncology Trials." JNCI: Journal of the National Cancer Institute 111, no. 12 (2019): 1255–62. http://dx.doi.org/10.1093/jnci/djz126.

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AbstractHistorically, phase II oncology trials assessed a treatment’s efficacy by examining its tumor response rate in a single-arm trial. Then, approximately 25 years ago, certain statistical and pharmacological considerations ignited a debate around whether randomized designs should be used instead. Here, based on an extensive literature review, we review the arguments on either side of this debate. In particular, we describe the numerous factors that relate to the reliance of single-arm trials on historical control data and detail the trial scenarios in which there was general agreement on

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Shi, Haolun, and Guosheng Yin. "Two-stage seamless transition design from open-label single-arm to randomized double-arm clinical trials." Statistical Methods in Medical Research 27, no. 1 (2016): 158–71. http://dx.doi.org/10.1177/0962280215625681.

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Conventional phase II clinical trials use either a single- or multi-arm comparison scheme to examine the therapeutic effects of the experimental drug. Both single- and multi-arm evaluations have their own merits; for example, single-arm phase II trials are easy to conduct and often require a smaller sample size, while multiarm trials are randomized and typically lead to a more objective comparison. To bridge the single- and double-arm schemes in one trial, we propose a two-stage design, in which the first stage takes a single-arm comparison of the experimental drug with the standard response r

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Mossop, Helen, Michael J. Grayling, Ferdia A. Gallagher, Sarah J. Welsh, Grant D. Stewart, and James M. S. Wason. "Advantages of multi-arm non-randomised sequentially allocated cohort designs for Phase II oncology trials." British Journal of Cancer 126, no. 2 (2021): 204–10. http://dx.doi.org/10.1038/s41416-021-01613-5.

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Abstract Background Efficient trial designs are required to prioritise promising drugs within Phase II trials. Adaptive designs are examples of such designs, but their efficiency is reduced if there is a delay in assessing patient responses to treatment. Methods Motivated by the WIRE trial in renal cell carcinoma (NCT03741426), we compare three trial approaches to testing multiple treatment arms: (1) single-arm trials in sequence with interim analyses; (2) a parallel multi-arm multi-stage trial and (3) the design used in WIRE, which we call the Multi-Arm Sequential Trial with Efficient Recruit

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Yin, Xiang, Ruthanna Davi, Elizabeth B. Lamont, et al. "Abstract 1025: Phase Ib trial single-arm efficacy estimates via comparison to a historical clinical trial synthetic control arm." Cancer Research 82, no. 12_Supplement (2022): 1025. http://dx.doi.org/10.1158/1538-7445.am2022-1025.

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Abstract Background: Innovations in data science and trial design have catalyzed novel clinical research methods which may inform the future success of new experimental therapies more efficiently than previously believed. To guide development of the novel immune agent GEN-1, we compared endpoints experienced by patients from a recent neoadjuvant single-arm phase Ib study of GEN-1 plus standard chemotherapy to those of similar historical clinical trial patients in receipt of standard chemotherapy alone. Methods: To compare safety and efficacy endpoints following first-line neoadjuvant weekly GE

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Hahn, Andreas, Ulrike Loderstädt, Hagen Frickmann, and Norbert G. Schwarz. "Sparing the control arm using well-characterized diagnostic approaches – the Gart and Buck prevalence estimator for efficacy estimation in single-arm trials." Journal of Laboratory Medicine 43, no. 5 (2019): 279–81. http://dx.doi.org/10.1515/labmed-2019-0091.

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Abstract Efficacy estimation of medical interventions in clinical trials requires diagnostics to assess the study endpoint. The imperfect nature of diagnostics often leads to biased efficacy estimations, usually underestimation, that could result in failure of clinical trials. Adjustment methods can be used if sensitivity and specificity are known, but they are not regularly applied. Double-arm clinical trials are the standard for demonstrating the superiority of a medical intervention. However, sometimes single-arm trials are the only option: for example, if a parallel group trial design with

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Alexander, Brian Michael, Patrick Y. Wen, Eudocia Quant Lee, et al. "Bayesian adaptive randomized trial design for patients with recurrent glioblastoma." Journal of Clinical Oncology 30, no. 15_suppl (2012): 2005. http://dx.doi.org/10.1200/jco.2012.30.15_suppl.2005.

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2005 Background: Bayesian-based trial design has the ability to utilize accumulating data in real time to alter the course of the trial, thereby enabling dynamic allocation to experimental arms and earlier dropping of ineffective arms. This flexibility results in a potentially more efficient trial framework by increasing the probability of enrollment to arms that show evidence of efficacy. In this study we considered a hypothetical scenario in which patients who have been previously treated on several separate experimental protocols at the Dana-Farber/Harvard Cancer Center and the University o

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Wu, Jianrong. "Single-arm Phase II cancer survival trial designs." Journal of Biopharmaceutical Statistics 26, no. 4 (2016): 644–56. http://dx.doi.org/10.1080/10543406.2015.1052494.

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Afshar, Afsheen, Gopal Santhanam, Byron M. Yu, Stephen I. Ryu, Maneesh Sahani, and Krishna V. Shenoy. "Single-Trial Neural Correlates of Arm Movement Preparation." Neuron 71, no. 3 (2011): 555–64. http://dx.doi.org/10.1016/j.neuron.2011.05.047.

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Thall, P. F., R. M. Simon, and E. H. Estey. "New statistical strategy for monitoring safety and efficacy in single-arm clinical trials." Journal of Clinical Oncology 14, no. 1 (1996): 296–303. http://dx.doi.org/10.1200/jco.1996.14.1.296.

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PURPOSE Efficacy and toxicity are both important outcomes in cancer clinical trials. Nonetheless, most statistical designs for phase II trials only provide rules for evaluating treatment efficacy, and moreover only allow early stopping after fixed cohorts of patients have been treated. We illustrate a new statistical design strategy for monitoring both adverse and efficacy outcomes on a patient-by-patient basis in phase II and other single-arm clinical trials. DESIGN The new strategy is used to design a phase II trial of the experimental regimen idarubicin plus cytarabine (ara-C) plus cyclospo

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Kanapuru, Bindu, Harpreet Singh, Janice Kim, and Paul Gustav Kluetz. "Patient-reported outcomes (PRO) in cancer trials submitted to the FDA from 2012-2015." Journal of Clinical Oncology 35, no. 15_suppl (2017): e14024-e14024. http://dx.doi.org/10.1200/jco.2017.35.15_suppl.e14024.

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e14024 Background: PRO measures are commonly assessed in cancer trials. We reviewed the PRO strategy, tools and trial designs for new drug applications (NDA) and biologics license applications (BLA) submitted to FDA over a 4 year period. Methods: A review of protocols and clinical study reports for original NDA and BLA applications submitted to the Office of Hematology and Oncology Products between 2012 and 2015 to support initial approval for adult malignant hematology and oncology conditions was done. We reviewed applications for inclusion of PRO data, trial design, type of PRO measure emplo

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Yin, Xiang, Elizabeth Stuart, Mehmet Burcu, Mark Stewart, Elizabeth Lamont, and Ruthanna Davi. "The Validity of a Synthetic Control Arm Derived from Historical Multiple Myeloma Clinical Trials." Blood 142, Supplement 1 (2023): 6628. http://dx.doi.org/10.1182/blood-2023-178892.

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Introduction While randomized phase III clinical trials (RCTs) are the gold-standard for establishing drug efficacy in the United States, external control arms (ECAs) may be valuable for both early and late phase drug development. An ECA is a collection of patients with the disease of interest who were treated outside of a clinical trial of interest (ie, target trial) and whose outcomes are compared to those of the target trial patients' to evaluate comparative drug efficacy and/or safety. ECAs created through rigorous statistical non-experimental methods such as matching patient-level charact

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Ray, Aniket Singha, Subhasish Ganguly, Shyamal Kumar Mukherjee, et al. "Homoeopathic Treatment in Hypothyroidism—An Open-Label, Prospective, Single-Arm, Clinical Trial." Homœopathic Links 34, no. 03 (2021): 191–98. http://dx.doi.org/10.1055/s-0041-1735489.

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Abstract Background Hypothyroidism is one of the most common endocrinal disorders in India and its incidence is gradually increasing. A preliminary clinical trial was conducted to assess the probable role of individualised homoeopathic (IH) medicines in hypothyroidism with the assessment of hormonal titre (serum thyroid-stimulating hormone [TSH] and free T4) and symptomatic improvement. Methods An open-label, prospective, single-arm trial was conducted on 40 patients suffering from hypothyroidism at the outpatient of D. N. De Homeopathic Medical College and Hospital, West Bengal, India, and tr

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Hsiue, Emily Han-Chung, Thomas J. Moore, and G. Caleb Alexander. "Estimated costs of pivotal trials for U.S. Food and Drug Administration–approved cancer drugs, 2015–2017." Clinical Trials 17, no. 2 (2020): 119–25. http://dx.doi.org/10.1177/1740774520907609.

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Background: Pivotal clinical trials provide critical evidence to regulators regarding a product’s suitability for marketing approval. The objectives of this study are (1) to characterize select features of trials for oncology products approved by the U.S. Food and Drug Administration between 2015 and 2017; and (2) to quantify the costs of these trials and how such costs varied based on trial characteristics. Methods: We identified novel oncology therapeutic drugs, and their respective pivotal trials, approved between 2015 and 2017 using annual summary reports from the Food and Drug Administrat

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Teramukai, Satoshi, Takashi Daimon, and Sarah Zohar. "A new design for phase II single-arm clinical trials: Bayesian predictive sample size selection design." Journal of Clinical Oncology 31, no. 15_suppl (2013): 6576. http://dx.doi.org/10.1200/jco.2013.31.15_suppl.6576.

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6576 Background: The aim of phase II trials is to determine if a new treatment is promising for further testing in confirmatory clinical trials. Most phase II clinical trials are designed as single-arm trials using a binary outcome with or without interim monitoring for early stopping. In this context, we propose a Bayesian adaptive design denoted as PSSD, predictive sample size selection design (Statistics in Medicine 2012;31:4243-4254). Methods: The design allows for sample size selection followed by any planned interim analyses for early stopping of a trial, together with sample size determ

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Gala, Kinisha, Ankit Kalucha, Samuel Martinet, et al. "Control arm heterogeneity in trials for unselected advanced triple-negative breast cancer (aTNBC)." Journal of Clinical Oncology 39, no. 15_suppl (2021): 1082. http://dx.doi.org/10.1200/jco.2021.39.15_suppl.1082.

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1082 Background: Primary endpoints of clinical trials frequently include subgroup-analyses. Several solid cancers such as aTNBC are heterogeneous, which can lead to unpredictable control arm performance impairing accurate assumptions for sample size calculations. We explore the value of a comprehensive clinical trial results repository in assessing control arm heterogeneity with aTNBC as the pilot. Methods: We identified P2/3 trials reporting median overall survival (mOS) and/or median progression-free survival (mPFS) in unselected aTNBC through a systematic search of PubMed, clinical trials d

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Pavel, Marianne, Dieter Hörsch, Martyn Caplin, et al. "Telotristat Etiprate for Carcinoid Syndrome: A Single-Arm, Multicenter Trial." Journal of Clinical Endocrinology & Metabolism 100, no. 4 (2015): 1511–19. http://dx.doi.org/10.1210/jc.2014-2247.

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Wu, Jianrong. "Single-arm phase II trial design under parametric cure models." Pharmaceutical Statistics 14, no. 3 (2015): 226–32. http://dx.doi.org/10.1002/pst.1678.

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Trippa, Lorenzo, Eudocia Q. Lee, Patrick Y. Wen, et al. "Bayesian Adaptive Randomized Trial Design for Patients With Recurrent Glioblastoma." Journal of Clinical Oncology 30, no. 26 (2012): 3258–63. http://dx.doi.org/10.1200/jco.2011.39.8420.

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Purpose To evaluate whether the use of Bayesian adaptive randomized (AR) designs in clinical trials for glioblastoma is feasible and would allow for more efficient trials. Patients and Methods We generated an adaptive randomization procedure that was retrospectively applied to primary patient data from four separate phase II clinical trials in patients with recurrent glioblastoma. We then compared AR designs with more conventional trial designs by using realistic hypothetical scenarios consistent with survival data reported in the literature. Our primary end point was the number of patients ne

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Gupta, Alind, Grace Hsu, Seamus Kent, et al. "Quantitative Bias Analysis for Single-Arm Trials With External Control Arms." JAMA Network Open 8, no. 3 (2025): e252152. https://doi.org/10.1001/jamanetworkopen.2025.2152.

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ImportanceUnmeasured confounding is a key concern for decision-makers when observational datasets are used to assemble external control arms (ECAs) for single-arm trials.ObjectiveTo investigate the utility of quantitative bias analysis (QBA) for exploring the sensitivity to unmeasured confounding of nonrandomized analyses using ECAs.Design, Setting, and ParticipantsThis study emulated 15 treatment comparisons using experimental arms from existing randomized trials in advanced non–small cell lung cancer (aNSCLC) conducted after 2011 and ECAs derived from observational data. Participants were el

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Tang, H., N. R. Foster, A. Grothey, S. M. Ansell, and D. J. Sargent. "Excessive false-positive errors in single-arm phase II trials: A simulation-based analysis." Journal of Clinical Oncology 27, no. 15_suppl (2009): 6512. http://dx.doi.org/10.1200/jco.2009.27.15_suppl.6512.

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6512 Background: The use of randomized phase II designs with an experimental arm and a standard-treatment control arm (R2PII) instead of a conventional single-arm design is clearly increasing in oncology. In practice, sample size, related cost issues, the belief that historical controls are adequate, and the use of a standard-treatment control arm in a phase II setting are frequently raised objections to R2PII trials. As the expense and complexity of definitive phase III trials increase, the ability of phase II trials to provide reliable and accurate results is critical. Methods: We investigat

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Keam, Bhumsuk, Eun Joo Kang, Myung-Ju Ahn, et al. "Randomized phase II study of axitinib versus observation in patients with recurred or metastatic adenoid cystic carcinoma." Journal of Clinical Oncology 38, no. 15_suppl (2020): 6503. http://dx.doi.org/10.1200/jco.2020.38.15_suppl.6503.

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6503 Background: Adenoid cystic carcinoma (ACC) does not respond to cytotoxic chemotherapy. Several anti-angiogenic agents were evaluated in single arm phase II trials. However, the role of chemotherapy is still controversial, because of natural stable disease course without chemotherapy and lack of randomized trial. We firstly conducted a randomized trial to evaluate the efficacy of axitinib compared to observation. Methods: In this multicenter, prospective phase II trial, we enrolled recurred, metastatic ACC patients who progressed within 9 months. Patients were randomly assigned either axit

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Yin, Xiang, Elizabeth Stuart, Mehmet Burcu, Mark Stewart, Elizabeth B. Lamont, and Ruthanna Davi. "Assessing the impact of unmeasured confounding in external control arms via tipping point analyses." Journal of Clinical Oncology 42, no. 16_suppl (2024): e23065-e23065. http://dx.doi.org/10.1200/jco.2024.42.16_suppl.e23065.

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e23065 Background: Estimates of the comparative efficacy of new therapies from single-arm settings can be obtained in advance of RCTs through use of external control arms (ECAs).[1] ECAs are collections of patients with the index disease who were treated outside of the single-arm trial, whose measured baseline attributes are matched to the single-arm trial patients and whose outcomes are compared to trial patients’ to estimate comparative efficacy. Without randomization, observed treatment-outcome associations may be confounded by unmeasured patient attributes. Applying established statistical

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Sun, Yuying, Samantha S. W. Fung, Patrick K. W. Man, et al. "Promoting Fruit and Vegetable Intake in Parents: A Cluster Randomised Controlled Trial." International Journal of Environmental Research and Public Health 18, no. 10 (2021): 5206. http://dx.doi.org/10.3390/ijerph18105206.

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We conducted a cluster randomised controlled trial of parents in 56 primary schools and community service centres (clusters) to evaluate the effectiveness of a single-session workshop on promoting more fruit and vegetable (FV) intake. A total of 803 parents were randomised to the FV intervention arm (16 clusters, n = 197), the more appreciation control arm (19 clusters, n = 270), or the less criticism control arm (21 clusters, n = 336). The FV intake of the FV arm was compared with that of the combined more appreciation or less criticism (MALC) arm. Both arms received a 2 h workshop: (i) the F

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Dholakiya, Dhaval. "Efficacy of Nirgundi Taila application in the management of Sandhigatavata (Osteoarthritis)- An open labelled single arm clinical trial." Journal of Research in Traditional Medicine 3, no. 1 (2017): 5–11. http://dx.doi.org/10.21276/jrtm.2017/460.

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Donchin, O., A. Gribova, O. Steinberg, A. R. Mitz, H. Bergman, and E. Vaadia. "Single-Unit Activity Related to Bimanual Arm Movements in the Primary and Supplementary Motor Cortices." Journal of Neurophysiology 88, no. 6 (2002): 3498–517. http://dx.doi.org/10.1152/jn.00335.2001.

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Single units were recorded from the primary motor (MI) and supplementary motor (SMA) areas of Rhesus monkeys performing one-arm (unimanual) and two-arm (bimanual) proximal reaching tasks. During execution of the bimanual movements, the task related activity of about one-half the neurons in each area (MI: 129/232, SMA: 107/206) differed from the activity during similar displacements of one arm while the other was stationary. The bulk of this “bimanual-related” activity could not be explained by any linear combination of activities during unimanual reaching or by differences in kinematics or rec

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Giotakis, A. I., E. M. Karow, M. O. Scheithauer, R. Weber, and H. Riechelmann. "Saline irrigations following sinus surgery - a controlled, single blinded, randomized trial." Rhinology journal 54, no. 4 (2016): 302–10. http://dx.doi.org/10.4193/rhino16.026.

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Background: Chronic rhinosinusitis (CRS) is a common health problem. If medical treatment fails, endonasal sinus surgery is a valuable treatment option. A thorough postsurgical treatment is needed including, among others, nasal saline irrigations (NSI). In this prospective, controlled, single blinded, randomized trial, we aimed to evaluate efficacy of nasal saline irrigations following endonasal sinus surgery in CRS-patients with nasal polyps. Methodology: We examined patient's nasal symptoms, general quality of life and postoperative condition of the mucosa. We also investigated whether or no

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Hashmi, Mustafa, Jeremy Rassen, and Sebastian Schneeweiss. "Single-arm oncology trials and the nature of external controls arms." Journal of Comparative Effectiveness Research 10, no. 12 (2021): 1053–66. http://dx.doi.org/10.2217/cer-2021-0003.

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Aim: Single-arm trials with external control arms (ECAs) have gained popularity in oncology. ECAs may consist of primary data from previous trials, electronic health records (EHRs) or aggregate data from the literature. We sought to provide a description of how such studies achieve similarity of patients, comparability of data quality and outcome assessment. Materials & methods: In a stratified convenience sample of 15 studies, five used primary data from trials as ECAs, five used secondary data from EHRs and five used aggregate data from the literature. Data were collected from the publis

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Renninson, Emily, Amarnath Challapalli, Emily Foulstone, et al. "A phase II trial of cemiplimab alone or in combination with standard of care chemotherapy in locally advanced or metastatic penile carcinoma (EPIC Trial)." Journal of Clinical Oncology 40, no. 16_suppl (2022): TPS5094. http://dx.doi.org/10.1200/jco.2022.40.16_suppl.tps5094.

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TPS5094 Background: Penile cancer (PC) is rare but its incidence is increasing, with an increase of 21% over the last decade. The rarity of this cancer means a paucity of large prospective clinical trial data to guide the management of locally advanced/metastatic penile cancer (la/mPC). Cisplatin containing combination chemotherapy regimens are widely regarded as the standard of care (SOC) in this setting. However, with response rates of about 50% there is a need to further improve treatment outcomes. PDL1 is upregulated in 40–60% of PC cases and is correlated with poor prognosis making a case

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Berry, Donald A., Michael Elashoff, Steven Blotner, et al. "Creating a synthetic control arm from previous clinical trials: Application to establishing early end points as indicators of overall survival in acute myeloid leukemia (AML)." Journal of Clinical Oncology 35, no. 15_suppl (2017): 7021. http://dx.doi.org/10.1200/jco.2017.35.15_suppl.7021.

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7021 Background: Clinical trials of experimental drugs require controls. Concurrently randomized controls are the gold standard for judging drug effect. Historical controls are not ideal but are much more efficient and economical. Historical controls derived from a single clinical trial have the biases of that trial. Using many trials with comparable end points and eligibility minimizes such bias. Medidata’s archive contains >3000 trials with clinical data rights for deidentified aggregated analyses. We used this resource to develop a synthetic control arm (SCA) for a particular phase I/II

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Chino, K., A. Shibata, A. Okuyama, et al. "THU0576 Tocilizumab Mono-Therapy for Polymyalgia Rheumatica ∼ A Single-Center, Open, Single-Arm Trial." Annals of the Rheumatic Diseases 75, Suppl 2 (2016): 400.2–400. http://dx.doi.org/10.1136/annrheumdis-2016-eular.1894.

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Lorenzi, Elizabeth, Amy M. Crawford, Craig S. Anderson, et al. "Adaptive Platform Trials in Stroke." Stroke 56, no. 1 (2025): 198–208. https://doi.org/10.1161/strokeaha.124.045754.

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Clinical trials of treatments for stroke have generally utilized 2-arm, randomized designs to evaluate a single intervention against a control. Running separate clinical trials, with each addressing a single therapeutic question, is resource intensive and slows evidence generation, especially in a field with rapidly expanding treatment options and evolving practices. Platform trials—randomized clinical trials designed to evaluate multiple interventions that may enter and exit the ongoing platform based on a master protocol—accelerate the investigation of multiple therapeutic options within a s

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Weijs, T. J., G. A. P. Nieuwenhuijzen, J. P. Ruurda, et al. "Study protocol for the nutritional route in oesophageal resection trial: a single-arm feasibility trial (NUTRIENT trial)." BMJ Open 4, no. 6 (2014): e004557-e004557. http://dx.doi.org/10.1136/bmjopen-2013-004557.

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Lloyd, Chris J. "Exact confidence limits after a group sequential single arm binary trial." Statistics in Medicine 40, no. 10 (2021): 2389–99. http://dx.doi.org/10.1002/sim.8909.

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Saleh, Parviz, Mohammad Naghavi-Behzad, Hamdieh Herizchi, Fatemeh Mokhtari, Mohammad Mirza-Aghazadeh-Attari, and Reza Piri. "Effects ofHelicobacter pyloritreatment on rosacea: A single-arm clinical trial study." Journal of Dermatology 44, no. 9 (2017): 1033–37. http://dx.doi.org/10.1111/1346-8138.13878.

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Ruggenenti, Piero, Erica Daina, Alessia Gennarini, et al. "C5 Convertase Blockade in Membranoproliferative Glomerulonephritis: A Single-Arm Clinical Trial." American Journal of Kidney Diseases 74, no. 2 (2019): 224–38. http://dx.doi.org/10.1053/j.ajkd.2018.12.046.

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Matshitsa, Lorato, Nassali Mercy-Nkuba, and G. Justus Hofmeyr. "Extended balloon labour induction: A single arm proof of concept trial." European Journal of Obstetrics & Gynecology and Reproductive Biology: X 19 (September 2023): 100226. http://dx.doi.org/10.1016/j.eurox.2023.100226.

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Matshitsa, Lorato, Nassali Mercy-Nkuba, and G. Justus Hofmeyr. "Extended balloon labour induction: A single arm proof of concept trial." European Journal of Obstetrics & Gynecology and Reproductive Biology: X 19 (September 2023): 100226. http://dx.doi.org/10.1016/j.eurox.2023.100226.

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Adelstein, David J., Yi Li, George L. Adams, et al. "An Intergroup Phase III Comparison of Standard Radiation Therapy and Two Schedules of Concurrent Chemoradiotherapy in Patients With Unresectable Squamous Cell Head and Neck Cancer." Journal of Clinical Oncology 21, no. 1 (2003): 92–98. http://dx.doi.org/10.1200/jco.2003.01.008.

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Purpose: The Head and Neck Intergroup conducted a phase III randomized trial to test the benefit of adding chemotherapy to radiation in patients with unresectable squamous cell head and neck cancer. Patients and Methods: Eligible patients were randomly assigned between arm A (the control), single daily fractionated radiation (70 Gy at 2 Gy/d); arm B, identical radiation therapy with concurrent bolus cisplatin, given on days 1, 22, and 43; and arm C, a split course of single daily fractionated radiation and three cycles of concurrent infusional fluorouracil and bolus cisplatin chemotherapy, 30

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Amr, A. Nimer, Michael Kosterhon, Eleftherios Archavlis, Florian Ringel, and Alexander L. Green. "113 Testing the Efficacy of Simulation in Neurosurgical Education: First Results of the SENSE Trial." Neurosurgery 64, CN_suppl_1 (2017): 223–24. http://dx.doi.org/10.1093/neuros/nyx417.113.

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Abstract INTRODUCTION Surgical education has hitherto relied heavily on the age-old acute;see one, do oneacute; paradigm. Factors such as traineesacute; decreased OR exposure due to worktime directives have put this paradigm to the test. The improvement of surgical skills in simulation laboratories has been touted as a possible solution and adopted in disciplines such as general surgery. We investigated the efficacy of a simulation curriculum on skill acquisition of a neurosurgical procedure (EVD-placement) by means of a single-blinded RCT. METHODS The Simulation Efficacy in Neurosurgical Educ

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Loprinzi, Charles L., Ralph Levitt, Debra Barton, et al. "Phase III Comparison of Depomedroxyprogesterone Acetate to Venlafaxine for Managing Hot Flashes: North Central Cancer Treatment Group Trial N99C7." Journal of Clinical Oncology 24, no. 9 (2006): 1409–14. http://dx.doi.org/10.1200/jco.2005.04.7324.

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Purpose Vasomotor hot flashes are a common problem in menopausal women. Given concerns regarding estrogen and/or combined hormonal therapy, other treatment options are desired. Prior trials have confirmed that progestational agents and newer antidepressants effectively reduce hot flashes. This current trial compared a single intramuscular dose of medroxyprogesterone acetate (MPA), depot preparation, versus daily oral venlafaxine as treatment for hot flashes. Methods Women with bothersome hot flashes were entered onto this trial, were randomly assigned to treatment, and then had a baseline week

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Grayling, Michael J., and Adrian P. Mander. "Two-Stage Single-Arm Trials Are Rarely Analyzed Effectively or Reported Adequately." JCO Precision Oncology, no. 5 (November 2021): 1813–20. http://dx.doi.org/10.1200/po.21.00276.

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PURPOSE Two-stage single-arm designs have historically been the most common design used in phase II oncology. They remain a mainstay today, particularly for trials in rare subgroups. Consequently, it is imperative such studies be designed, analyzed, and reported effectively. We comprehensively review such trials to examine whether this is the case. METHODS Oncology trials that used Simon's two-stage design over a 5-year period were identified and reviewed. They were evaluated for whether they reported sufficient design (eg, required sample size) and analysis (eg, CI) details. Articles that did

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Rai, Ansaar T., Thabele M. Leslie-Mazwi, Kyle M. Fargen, et al. "Neuroendovascular clinical trials disruptions due to COVID-19. Potential future challenges and opportunities." Journal of NeuroInterventional Surgery 12, no. 9 (2020): 831–35. http://dx.doi.org/10.1136/neurintsurg-2020-016502.

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To assess the impact of COVID-19 on neurovascular research and deal with the challenges imposed by the pandemic.MethodsA survey-based study focused on randomized controlled trials (RCTs) and single-arm studies for acute ischemic stroke and cerebral aneurysms was developed by a group of senior neurointerventionalists and sent to sites identified through the clinical trials website (https://clinicaltrials.gov/), study sponsors, and physician investigators.ResultsThe survey was sent to 101 institutions, with 65 responding (64%). Stroke RCTs were being conducted at 40 (62%) sites, aneurysm RCTs at

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Viele, Kert, Kristine Broglio, Anna McGlothlin, and Benjamin R. Saville. "Comparison of methods for control allocation in multiple arm studies using response adaptive randomization." Clinical Trials 17, no. 1 (2019): 52–60. http://dx.doi.org/10.1177/1740774519877836.

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Background/Aims: Response adaptive randomization has many polarizing properties in two-arm settings comparing control to a single treatment. The generalization of these features to the multiple arm setting has been less explored, and existing comparisons in the literature reach disparate conclusions. We investigate several generalizations of two-arm response adaptive randomization methods relating to control allocation in multiple arm trials, exploring how critiques of response adaptive randomization generalize to the multiple arm setting. Methods: We perform a simulation study to investigate

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Santoro, A., T. Tursz, H. Mouridsen, et al. "Doxorubicin versus CYVADIC versus doxorubicin plus ifosfamide in first-line treatment of advanced soft tissue sarcomas: a randomized study of the European Organization for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group." Journal of Clinical Oncology 13, no. 7 (1995): 1537–45. http://dx.doi.org/10.1200/jco.1995.13.7.1537.

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PURPOSE The aim of this trial was to compare the activity and toxicity of single-agent doxorubicin with that of two multidrug regimens in the treatment of patients with adult advanced soft tissue sarcomas. PATIENTS AND METHODS This was a prospective randomized phase III trial performed by 35 cancer centers within the Soft Tissue and Bone Sarcoma Group of the European Organization for Research and Treatment of Cancer (EORTC). Six hundred sixty-three eligible patients were randomly allocated to receive either doxorubicin 75 mg/m2 (arm A), cyclophosphamide, vincristine, doxorubicin, and dacarbazi

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Mazor, Tali, Karim S. Farhat, Pavel Trukhanov, et al. "Clinical Trial Notifications Triggered by Artificial Intelligence–Detected Cancer Progression." JAMA Network Open 8, no. 4 (2025): e252013. https://doi.org/10.1001/jamanetworkopen.2025.2013.

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ImportanceHistorically, fewer than 10% of adults with cancer have enrolled in clinical trials. Computational tools have been developed to match patients to trials, but these tools are relevant only when patients need new treatment.ObjectiveTo evaluate whether notifying oncologists about genomically targeted clinical trials for patients with cancer progression, as detected by artificial intelligence (AI), impacts clinical trial participation.Design, Setting, and ParticipantsThis single-center randomized trial was conducted from January 30, 2023, to June 30, 2024, at a tertiary academic cancer c

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Boye, Kjetil, Alessandra Longhi, Tormod Guren, et al. "Pembrolizumab in advanced osteosarcoma: results of a single-arm, open-label, phase 2 trial." Cancer Immunology, Immunotherapy 70, no. 9 (2021): 2617–24. http://dx.doi.org/10.1007/s00262-021-02876-w.

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Abstract Aim To evaluate the activity and safety of the PD-1 antibody pembrolizumab in adult patients with advanced osteosarcoma. Material and methods The study was a single-arm, open-label, phase 2 trial in patients with unresectable, relapsed osteosarcoma. The primary endpoint was clinical benefit rate (CBR) at 18 weeks of treatment, defined as complete response, partial response, or stable disease using RECIST v1.1. The trial had a Simon´s two-stage design, and ≥ 3 of 12 patients with clinical benefit in stage 1 were required to proceed to stage 2. The trial is registered with ClinicalTrial

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Hao, Yanni, Wei-Chun Hsu, Craig S. Parzynski, et al. "Utilization of External Control Arm to Contextualize Clinical Efficacy of Tisagenlecleucel Treated Among Patients with Relapsed/Refractory Follicular Lymphoma from the Single-Arm Elara Trial." Blood 138, Supplement 1 (2021): 4506. http://dx.doi.org/10.1182/blood-2021-152685.

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Abstract Introduction: While randomized clinical trials (RCT) remain the gold standard for evaluating the safety and efficacy of treatments, interest in the use of external control arm (ECA) has grown when randomization is either infeasible or unethical (Friends of Cancer Research 2019). ECAs can provide contextual evidence to support regulatory approval in such situations. Single arm trials in patients with relapsed/refractory follicular lymphoma (FL) is one example due to the rarity of the disease and lack of standardized treatment in this patient population. The target trial framework (Hern

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