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1

Chi, George Y. H. "Multiple Testings: Multiple Comparisons and Multiple Endpoints." Drug Information Journal 32, no. 1_suppl (1998): 1347S—1362S. http://dx.doi.org/10.1177/00928615980320s131.

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2

Dueck, Amylou, Paul J. Novotny, and Jeff A. Sloan. "Dealing with Multiple Endpoints." Current Problems in Cancer 30, no. 6 (2006): 298–306. http://dx.doi.org/10.1016/j.currproblcancer.2006.08.007.

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3

Häberle, Lothar, Annette Pfahlberg, and Olaf Gefeller. "Assessment of Multiple Ordinal Endpoints." Biometrical Journal 51, no. 1 (2009): 217–26. http://dx.doi.org/10.1002/bimj.200810502.

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4

Hasler, Mario, and Kathrin Böhlendorf. "Multiple Comparisons for Multiple Endpoints in Agricultural Experiments." Journal of Agricultural, Biological, and Environmental Statistics 18, no. 4 (2013): 578–93. http://dx.doi.org/10.1007/s13253-013-0149-7.

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5

Tsai, Kao-Tai, and James A. Kozial. "Assessing multiple endpoints with ordered alternatives." Communications in Statistics - Theory and Methods 23, no. 2 (1994): 533–44. http://dx.doi.org/10.1080/03610929408831270.

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6

Xu, Jane, and Scott L. Zeger. "The Evaluation of Multiple Surrogate Endpoints." Biometrics 57, no. 1 (2001): 81–87. http://dx.doi.org/10.1111/j.0006-341x.2001.00081.x.

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7

Eaton, Morris L., and Robb J. Muirhead. "On a multiple endpoints testing problem." Journal of Statistical Planning and Inference 137, no. 11 (2007): 3416–29. http://dx.doi.org/10.1016/j.jspi.2007.03.021.

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8

Quan, Hui, Jim Bolognese, and Weiying Yuan. "Assessment of equivalence on multiple endpoints." Statistics in Medicine 20, no. 21 (2001): 3159–73. http://dx.doi.org/10.1002/sim.985.

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9

Ishihara, Takuma, and Kouji Yamamoto. "A Test for Multiple Binary Endpoints with Continuous Latent Distribution in Clinical Trials." Journal of Statistical Theory and Applications 20, no. 4 (2021): 463–80. http://dx.doi.org/10.1007/s44199-021-00003-3.

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AbstractIn clinical trials, two or more binary responses obtained by dichotomizing continuous responses are often employed as multiple primary endpoints. Testing procedures for multiple binary variables with latent distribution have not yet been adequately discussed. Based on the association measure among latent variables, we provide a statistic for testing the superiority of at least one binary endpoint. In addition, we propose a testing procedure with a framework in which the trial efficacy is confirmed only when there is superiority of at least one endpoint and non-inferiority of the remain
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10

Wei, Xinmiao, Tengxin Huang, Zhijiang Yang, Li Pan, Liangliang Wang, and Junjie Ding. "Quantitative Predictive Studies of Multiple Biological Activities of TRPV1 Modulators." Molecules 29, no. 2 (2024): 295. http://dx.doi.org/10.3390/molecules29020295.

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TRPV1 channel agonists and antagonists, which have powerful analgesic effects without the addictive qualities associated with traditional analgesics, have become a focus area for the development of novel analgesics. In this study, quantitative structure–activity relationship (QSAR) models for three bioactive endpoints (Ki, IC50, and EC50) were successfully constructed using four machine learning algorithms: SVM, Bagging, GBDT, and XGBoost. These models were based on 2922 TRPV1 modulators and incorporated four types of molecular descriptors: Daylight, E-state, ECFP4, and MACCS. After the rigoro
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11

Candida Fratazzi and Jixiao Niu. "Accelerated orphan drug approval: surrogate endpoints." World Journal of Advanced Pharmaceutical and Medical Research 2, no. 1 (2022): 001–7. http://dx.doi.org/10.53346/wjapmr.2022.2.1.0021.

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Today, orphan drug development is confronted with significant challenges represented by the considerable complexity, diversity of clinical manifestations, and competition in study recruitment. Thus, surrogate endpoints adoption plays a crucial role in rare disease trials by minimizing costs, the number of subjects, and study duration. Surrogate endpoints, to substitute for a direct measure of how patients feel, function, or survive, must be biomarkers that directly correlate with disease clinical manifestations and predict the impact of study drug on the long-term disease progression. Validati
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12

Jin, Man, and Pingye Zhang. "An adaptive seamless Phase 2-3 design with multiple endpoints." Statistical Methods in Medical Research 30, no. 4 (2021): 1143–51. http://dx.doi.org/10.1177/0962280220986935.

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Adaptive seamless Phase 2-3 design has been considered as one possible way to expedite development time for a drug program by allowing the expansion from an ongoing Phase 2 trial into a Phase 3 trial. Multiple endpoints are often tested when a regulatory approval is pursued. Here we propose an adaptive seamless Phase 2-3 design with multiple endpoints which can expand an ongoing Phase 2 trial into a Phase 3 trial based on an intermediate endpoint for adaptive decision and test the endpoints with a powerful multiple test procedure. It is proved that the proposed design can preserve the familywi
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13

Santosh Kumar, Rada, and A. Rama Krishna. "Clinical Endpoint: Substitute for Prediction of Clinical Benefit." Journal of Drug Delivery and Therapeutics 9, no. 4-s (2019): 800–802. http://dx.doi.org/10.22270/jddt.v9i4-s.3367.

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An endpoint is a primary or secondary outcome used to judge the effectiveness of a treatment; it is a precisely defined variable intended to reflect an outcome of interest thatis statistically analyzed. An endpoint usually specifies the sort of assessments made, the timing of those assessments, the assessment tools used and possibly other details, as applicable, such as how multiple assessments within an individual are to be combined. There are different types of endpoints used in clinical trails like primary endpoint, secondary endpoint, multiple endpoint and surrogate endpoint. Primary endpo
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14

Baracaldo-Santamaría, Daniela, John Edwin Feliciano-Alfonso, Raul Ramirez-Grueso, Luis Carlos Rojas-Rodríguez, Camilo Alberto Dominguez-Dominguez, and Carlos Alberto Calderon-Ospina. "Making Sense of Composite Endpoints in Clinical Research." Journal of Clinical Medicine 12, no. 13 (2023): 4371. http://dx.doi.org/10.3390/jcm12134371.

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Multiple drugs currently used in clinical practice have been approved by regulatory agencies based on studies that utilize composite endpoints. Composite endpoints are appealing because they reduce sample size requirements, follow-up periods, and costs. However, interpreting composite endpoints can be challenging, and their misuse is not uncommon. Incorrect interpretation of composite outcomes can lead to misleading conclusions that impact patient care. To correctly interpret composite outcomes, several important questions should be considered. Are the individual components of the composite ou
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15

Kavia, RBC, D. De Ridder, CS Constantinescu, CG Stott, and CJ Fowler. "Randomized controlled trial of Sativex to treat detrusor overactivity in multiple sclerosis." Multiple Sclerosis Journal 16, no. 11 (2010): 1349–59. http://dx.doi.org/10.1177/1352458510378020.

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Background: Bladder dysfunction is a common feature of multiple sclerosis (MS). Objective: In this study we aimed to assess the efficacy, tolerability and safety of Sativex® (nabiximols) as an add-on therapy in alleviating bladder symptoms in patients with MS. Methods: We undertook a 10-week, double-blind, randomized, placebo-controlled, parallel-group trial in 135 randomized subjects with MS and overactive bladder (OAB). Results: The primary endpoint was the reduction in daily number of urinary incontinence episodes from baseline to end of treatment (8 weeks). Other endpoints included inciden
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16

Chen, James J. "P-value adjustment for multiple binary endpoints." Communications in Statistics - Theory and Methods 27, no. 11 (1998): 2791–806. http://dx.doi.org/10.1080/03610929808832255.

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17

Whitehouse, C. A., A. M. Bothwell, E. J. Tawn, and R. Wood. "Radiation Dose Estimation Using Multiple Cytogenetic Endpoints." Radiation Protection Dosimetry 72, no. 2 (1997): 127–30. http://dx.doi.org/10.1093/oxfordjournals.rpd.a032082.

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18

Ristl, Robin, Dong Xi, Ekkehard Glimm, and Martin Posch. "Optimal exact tests for multiple binary endpoints." Computational Statistics & Data Analysis 122 (June 2018): 1–17. http://dx.doi.org/10.1016/j.csda.2018.01.001.

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19

Hasler, Mario, and Ludwig A. Hothorn. "A Dunnett-Type Procedure for Multiple Endpoints." International Journal of Biostatistics 7, no. 1 (2011): 1–15. http://dx.doi.org/10.2202/1557-4679.1258.

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20

Montgomery, Robert N., and Jonathan D. Mahnken. "A prediction‐based test for multiple endpoints." Statistics in Medicine 39, no. 28 (2020): 4267–80. http://dx.doi.org/10.1002/sim.8724.

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21

Wang, Yueyue, Crystal Haskell-Ramsay, Jose Lara Gallegos, and John K. Lodge. "Inter-Individual Responses to a Blueberry Intervention across Multiple Endpoints." Nutrients 16, no. 6 (2024): 895. http://dx.doi.org/10.3390/nu16060895.

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Inter-individual variation exists in response to diet and in the endpoints related to vascular diseases and cognitive impairment. Therefore, the evaluation and characterisation of responses to a dietary intervention targeting these endpoints is important. A dietary intervention with 37 participants has been performed comparing two forms of blueberry, either whole fresh blueberry (160 g), freeze-dried blueberry powder (20 g) or a placebo control (microcrystalline cellulose), in a 1-week single-blinded cross-over randomised controlled trial (RCT) in a healthy population. The response to the inte
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22

Ashall, Vanessa. "Endpoint Matrix: a conceptual tool to promote consideration of the multiple dimensions of humane endpoints." ALTEX 31, no. 2 (2014): 209–13. http://dx.doi.org/10.14573/altex.1307261.

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23

Bar-Zohar, D., F. Agosta, D. Goldstaub, and M. Filippi. "Magnetic resonance imaging metrics and their correlation with clinical outcomes in multiple sclerosis: a review of the literature and future perspectives." Multiple Sclerosis Journal 14, no. 6 (2008): 719–27. http://dx.doi.org/10.1177/1352458507088102.

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Magnetic resonance imaging (MRI) has revolutionized the diagnosis and management of patients with multiple sclerosis (MS). Conventional MRI metrics are employed as primary endpoints in proof-of-concept clinical trials evaluating new drugs for MS and as secondary endpoints in definitive phase III trials. Metrics derived from non-conventional MRI techniques are now emerging and hold significant promise since they appear to be more correlated with the most disabling features of MS. However, none of these has been approved for use as a surrogate endpoint for accumulation of physical disability, wh
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24

Lavery, Amy M., Leonard H. Verhey, and Amy T. Waldman. "Outcome Measures in Relapsing-Remitting Multiple Sclerosis: Capturing Disability and Disease Progression in Clinical Trials." Multiple Sclerosis International 2014 (2014): 1–13. http://dx.doi.org/10.1155/2014/262350.

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Multiple sclerosis (MS) is a chronic inflammatory and neurodegenerative disease that manifests as acute relapses and progressive disability. As a primary endpoint for clinical trials in MS, disability is difficult to both characterize and measure. Furthermore, the recovery from relapses and the rate of disability vary considerably among patients. Given these challenges, investigators have developed and studied the performance of various outcome measures and surrogate endpoints in MS clinical trials. This review defines the outcome measures and surrogate endpoints used to date in MS clinical tr
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25

Kieser, M., and G. Rauch. "Multiplicity Adjustment for Composite Binary Endpoints." Methods of Information in Medicine 51, no. 04 (2012): 309–17. http://dx.doi.org/10.3414/me11-01-0044.

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SummaryBackground: Binary composite outcome measures are increasingly used as primary endpoints in clinical trials. Composite endpoints combine several events of interest within a single variable. However, as the effect observed for the composite does not necessarily reflect the effects for the individual components, it is recommended in the literature to additionally evaluate each component separately.Objectives: The task is to define an adequate multiple test procedure which focuses on the composite outcome measure but allows for a confirmatory interpretation of the components in case of lar
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26

Rauch, Geraldine, and Meinhard Kieser. "Adaptive designs for clinical trials with multiple endpoints." Clinical Investigation 5, no. 5 (2015): 433–35. http://dx.doi.org/10.4155/cli.14.138.

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27

Pocock, Stuart J., Nancy L. Geller, and Anastasios A. Tsiatis. "The Analysis of Multiple Endpoints in Clinical Trials." Biometrics 43, no. 3 (1987): 487. http://dx.doi.org/10.2307/2531989.

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28

Mor, Maria K., and Stewart J. Anderson. "A Bayesian Group Sequential Approach for Multiple Endpoints." Sequential Analysis 24, no. 1 (2005): 45–62. http://dx.doi.org/10.1081/sqa-200046829.

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29

Chang, Mark, and Shein-Chung Chow. "Analysis Strategies for Adaptive Designs with Multiple Endpoints." Journal of Biopharmaceutical Statistics 17, no. 6 (2007): 1189–200. http://dx.doi.org/10.1080/10543400701645348.

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30

Van Der Schaaf, A., C. Schilstra, J. A. Langendijk, and A. van't Veld. "DATA-EFFICIENCY OF NTCP MODELLING USING MULTIPLE ENDPOINTS." Radiotherapy and Oncology 92 (August 2009): S240. http://dx.doi.org/10.1016/s0167-8140(12)73234-6.

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31

Kiyosawa, Naoki, Yosuke Ando, Kyoko Watanabe, Noriyo Niino, Sunao Manabe, and Takashi Yamoto. "Scoring multiple toxicological endpoints using a toxicogenomic database." Toxicology Letters 188, no. 2 (2009): 91–97. http://dx.doi.org/10.1016/j.toxlet.2009.03.011.

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32

Walker, A. Sarah, and Abdel G. Babiker. "S36 Multiple endpoints en trials en HIV infection." Controlled Clinical Trials 18, no. 3 (1997): S63. http://dx.doi.org/10.1016/s0197-2456(97)91021-7.

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33

Gönen, Mithat, Peter H. Westfall, and Wesley O. Johnson. "Bayesian Multiple Testing for Two-Sample Multivariate Endpoints." Biometrics 59, no. 1 (2003): 76–82. http://dx.doi.org/10.1111/1541-0420.00009.

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34

Shi, Qian, Emily S. Pavey, and Rickey E. Carter. "Bonferroni-based correction factor for multiple, correlated endpoints." Pharmaceutical Statistics 11, no. 4 (2012): 300–309. http://dx.doi.org/10.1002/pst.1514.

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35

Follmann, Dean. "Multivariate tests for multiple endpoints in clinical trials." Statistics in Medicine 14, no. 11 (1995): 1163–75. http://dx.doi.org/10.1002/sim.4780141103.

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36

Wang, Bushi, and Xinping Cui. "A new partition testing strategy for multiple endpoints." Statistics in Medicine 31, no. 20 (2012): 2151–68. http://dx.doi.org/10.1002/sim.5366.

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37

Belaunzaran, Miguel, Shahm Raslan, Aleeza Ali, Kevin Newsome, Mark McKenney, and Adel Elkbuli. "Utilization and Efficacy of Resuscitation Endpoints in Trauma and Burn Patients: A Review Article." American Surgeon 88, no. 1 (2021): 10–19. http://dx.doi.org/10.1177/00031348211060424.

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Shock is a sequelae in trauma and burn patients that substantially increases the risk for morbidity and mortality. The use of resuscitation endpoints allows for improved management of these patients, with the potential to prevent further morbidity/mortality. We conducted a review of the current literature on the efficacy of hemodynamic, metabolic, and regional resuscitation endpoints for use in trauma and burn patients. Hemodynamic endpoints included mean arterial pressure (MAP), heart rate (HR), urinary output (UO), compensatory reserve index (CRI), intrathoracic blood volume, and stroke volu
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38

Stoddard, Josh, Adam Mustafa, and Naveen Goela. "Tanium reveal." Proceedings of the VLDB Endowment 14, no. 12 (2021): 3096–109. http://dx.doi.org/10.14778/3476311.3476386.

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Tanium Reveal is a federated search engine deployed on large-scale enterprise networks that is capable of executing data queries across billions of private data files within 60 seconds. Data resides at the edge of networks, potentially distributed on hundreds of thousands of endpoints. The anatomy of the search engine consists of local inverse indexes on each endpoint and a global communication platform called Tanium for issuing search queries to all endpoints. Reveal enables asynchronous parsing and indexing on endpoints without noticeable impact to the endpoints' primary functionality. The e
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39

Revankar, Sanjay G., William R. Kirkpatrick, Robert K. McAtee, et al. "Interpretation of Trailing Endpoints in Antifungal Susceptibility Testing by the National Committee for Clinical Laboratory Standards Method." Journal of Clinical Microbiology 36, no. 1 (1998): 153–56. http://dx.doi.org/10.1128/jcm.36.1.153-156.1998.

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Trailing endpoints remain a problem in antifungal susceptibility testing using the National Committee for Clinical Laboratory Standards (NCCLS) method. For isolates for which trailing endpoints are found, MICs of ≤1 μg/ml at 24 h and of >64 μg/ml at 48 h are usually observed. In a study of human immunodeficiency virus (HIV)-infected patients with oropharyngeal candidiasis, we identified three patients with multiple serial isolates for which trailing endpoints were observed with fluconazole. At 24 h, MICs were generally ≤1 μg/ml by both broth macro- and microdilution methods. However, at 48
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40

Legault, Claudine. "68A Analyzing multiple endpoints from multiple time periods with multiple groups: The PEPI cohort." Controlled Clinical Trials 16, no. 3 (1995): 71S. http://dx.doi.org/10.1016/0197-2456(95)90479-o.

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41

Ashall, Vanessa. "Erratum to Endpoint Matrix: a Conceptual tool to Promote Consideration of the Multiple Dimensions of Humane Endpoints." ALTEX 31, no. 4 (2014): 422. http://dx.doi.org/10.14573/altex.1409081.

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42

Sun, Hengrui, Ellen Snyder, and Gary G. Koch. "Statistical planning in confirmatory clinical trials with multiple treatment groups, multiple visits, and multiple endpoints." Journal of Biopharmaceutical Statistics 28, no. 1 (2017): 189–211. http://dx.doi.org/10.1080/10543406.2017.1378664.

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43

Elser, C., G. Pond, E. Chen, and L. Siu. "A review of 565 phase II trial abstracts from ASCO 2005: Design characteristics of contemporary trials evaluating cytotoxic versus non-cytotoxic regimens." Journal of Clinical Oncology 25, no. 18_suppl (2007): 6569. http://dx.doi.org/10.1200/jco.2007.25.18_suppl.6569.

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6569 Background: New phase II trial designs have been proposed to accommodate the non-cytotoxic nature of molecularly targeted agents. In particular, the use of alternative endpoints and controls has been suggested. The objectives of this survey are to review the designs of contemporary phase II trials, to compare trials of cytotoxic versus non-cytotoxic regimens, and to identify predictors of acceptance for presentation. Methods: Abstracts of phase II trials of systemic anticancer therapy published in the 2005 ASCO Proceedings were hand searched. The trial context, type of intervention, trial
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44

Sozu, Takashi, Tomoyuki Sugimoto, and Toshimitsu Hamasaki. "Statistical Issues in Clinical Trials with Multiple Primary Endpoints." Japanese Journal of Biometrics 34, no. 1 (2013): 35–52. http://dx.doi.org/10.5691/jjb.34.35.

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45

Tang, Dei-In, Clare Gnecco, and Nancy L. Geller. "Design of Group Sequential Clinical Trials with Multiple Endpoints." Journal of the American Statistical Association 84, no. 407 (1989): 776. http://dx.doi.org/10.2307/2289665.

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46

Bloch, Daniel A., Tze Leung Lai, and Pascale Tubert-Bitter. "One-Sided Tests in Clinical Trials with Multiple Endpoints." Biometrics 57, no. 4 (2001): 1039–47. http://dx.doi.org/10.1111/j.0006-341x.2001.01039.x.

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47

Perlman, Michael D., and Lang Wu. "A Note on One‐Sided Tests with Multiple Endpoints." Biometrics 60, no. 1 (2004): 276–80. http://dx.doi.org/10.1111/j.0006-341x.2004.00159.x.

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48

Tang, Dei-In, Clare Gnecco, and Nancy L. Geller. "Design of Group Sequential Clinical Trials with Multiple Endpoints." Journal of the American Statistical Association 84, no. 407 (1989): 775–79. http://dx.doi.org/10.1080/01621459.1989.10478836.

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49

Sun, Anna, Xiaoyu Dong, and Yi Tsong. "Sample Size Determination for Equivalence Assessment with Multiple Endpoints." Journal of Biopharmaceutical Statistics 24, no. 6 (2014): 1203–14. http://dx.doi.org/10.1080/10543406.2014.941986.

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50

Neuhäuser, Markus. "How to deal with multiple endpoints in clinical trials." Fundamental and Clinical Pharmacology 20, no. 6 (2006): 515–23. http://dx.doi.org/10.1111/j.1472-8206.2006.00437.x.

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