Fundamentals of Clinical Trials

Clinical Trials: A Practical Approach.

Hill, I. D., & Pocock, S. (1984) · Biometrics · doi:10.2307/2531186 · Cited by 2,918 · see citing works

Designing Clinical Research: An Epidemiologic Approach

Hulley, S., Cummings, S., & Browner, W. (1988) · Controlled Clinical Trials · doi:10.1016/0197-2456(89)90023-8 · Cited by 1,043 · see citing works

Clinical Trials : A Methodologic Perspective

Piantadosi, S. (1997) · Cited by 803 · see citing works

Biostatistics in Clinical Medicine.

Brown, R. A., Ingelfinger, J. A., Mosteller, F. C., Thibodeau, L. A., & Ware, J. H. (1988) · Biometrics · doi:10.2307/2531609 · Cited by 329 · see citing works

Clinical Trials: Design, Conduct, and Analysis

Baker, L. H. (1987) · JAMA · doi:10.1001/jama.1987.03390090119041 · Cited by 231 · see citing works

Clinical Trials: Issues and Approaches.

Fleiss, J. L., Shapiro, S. H., & Louis, T. (1984) · Journal of the American Statistical Association · doi:10.2307/2288302 · Cited by 112 · see citing works

Introduction to Statistical Methods for Clinical Trials

Cook, T. D., & DeMets, D. L. (2007) · Chapman & Hall/CRC eBooks · doi:10.1201/9781420009965 · Cited by 96 · see citing works

Statistical Issues in Drug Research and Development.

Smith, M. K., & Peace, K. E. (1991) · Journal of the Royal Statistical Society Series C (Applied Statistics) · doi:10.2307/2347927 · Cited by 86 · see citing works

Clinical Trials in Oncology

Green, S., Benedetti, J., & Benedetti, J. (2012) · CRC Press eBooks · doi:10.1201/b12125 · Cited by 78 · see citing works

The Randomized Clinical Trial and Therapeutic Decisions.

McPherson, K., Tygstrup, N., Lachin, J. M., & Juhl, E. (1983) · Biometrics · doi:10.2307/2531034 · Cited by 70 · see citing works

Textbook of Clinical Trials

(2006) · Wiley eBooks · doi:10.1002/9780470010167 · Cited by 49 · see citing works

Randomised Controlled Clinical Trials

Bulpitt, C. J. (1983) · Springer eBooks · doi:10.1007/978-1-4757-6358-4 · Cited by 47 · see citing works

Advances in Clinical Trial Biostatistics

(2003) · CRC Press eBooks · doi:10.1201/9780203912881 · Cited by 42 · see citing works

Statistics in Medical Research: Methods and Issues with Applications in Cancer Research.

Metter, G. E., Miké, V., & Stanley, K. (1984) · Journal of the American Statistical Association · doi:10.2307/2288304 · Cited by 39 · see citing works

Statistics in Drug Research: Methodologies and Recent Developments

Chow, S., & Shao, J. (2002) · Cited by 31 · see citing works

Assessment of Health-Related Quality of Life

Friedman, L. M., Furberg, C. D., & DeMets, D. L. (2010) · Springer eBooks · doi:10.1007/978-1-4419-1586-3_13 · Cited by 28 · see citing works

Assessment of Health-Related Quality of Life

Naughton, M. J., & Shumaker, S. A. (1998) · Springer eBooks · doi:10.1007/978-1-4757-2915-3_12 · Cited by 24 · see citing works

Conducting Clinical Trials

Iber, F. L., Riley, W. A., & Murray, P. J. (1987) · Springer eBooks · doi:10.1007/978-1-4613-1919-1 · Cited by 23 · see citing works

Methodology of Clinical Drug Trials.

D’Agostino, R. B., Spriet, A., Dupin-Spriet, T., Simon, P., Coluzzi, R., Young, J., Edelstein, R., & WEINTRAUB, M. I. (1995) · Journal of the American Statistical Association · doi:10.2307/2291173 · Cited by 19 · see citing works

The Randomization Process

Friedman, L. M., Furberg, C. D., DeMets, D. L., Reboussin, D. M., & Granger, C. B. (2015) · Springer eBooks · doi:10.1007/978-3-319-18539-2_6 · Cited by 13 · see citing works

Response Adaptive Randomization

Coad, D. S. (2008) · Wiley Encyclopedia of Clinical Trials · doi:10.1002/9780471462422.eoct308 · Cited by 4 · see citing works

Conducting clinical trials

Hanauer, S. B. (1988) · Gastroenterology · doi:10.1016/0016-5085(88)90329-0 · Cited by 3 · see citing works

Disclosure of Conflict of Interest

(2021) · The Breast · doi:10.1016/s0960-9776(21)00583-x · Cited by 1 · see citing works

Multiplicity in randomised trials I: endpoints and treatments

Schulz, K. F., & Grimes, D. A. (2005) · The Lancet · doi:10.1016/s0140-6736(05)66461-6 · Cited by 366 · see citing works

Approaches to sample size estimation in the design of clinical trials—a review

DONNER, A. P. (1984) · Statistics in Medicine · doi:10.1002/sim.4780030302 · Cited by 250 · see citing works

Transfusion‐associated cancer recurrence and postoperative infection: meta‐analysis of randomized, controlled clinical trials

Vamvakas, E. C. (1996) · Transfusion · doi:10.1046/j.1537-2995.1996.36296181932.x · Cited by 207 · see citing works

Clinical outcomes in reduction mammaplasty: a systematic review and meta-analysis of published studies

Chadbourne, E., Zhang, S., Gordon, M. J., Ro, E. Y., Ross, S. D., Schnur, P., & Schneider-Redden, P. R. (2001) · Mayo Clinic Proceedings · doi:10.4065/76.5.503 · Cited by 166 · see citing works

Sequential Treatment Allocation Using Biased Coin Designs

Smith, R. L. (1984) · Journal of the Royal Statistical Society Series B (Statistical Methodology) · doi:10.1111/j.2517-6161.1984.tb01323.x · Cited by 122 · see citing works

Efficacy of Transsphenoidal Hypophysectomy in Treatment of Dogs with Pituitary-Dependent Hyperadrenocorticism

Hanson, J., Hoofd, M. M. V. ‘., Voorhout, G., Teske, E., Kooistra, H. S., & Meij, B. P. (2005) · Journal of Veterinary Internal Medicine · doi:10.1111/j.1939-1676.2005.tb02747.x · Cited by 97 · see citing works

A group sequential, response-adaptive design for randomized clinical trials

Karrison, T. G., Huo, D., & Chappell, R. J. (2003) · Controlled Clinical Trials · doi:10.1016/s0197-2456(03)00092-8 · Cited by 95 · see citing works

Maximizing power and minimizing treatment failures in clinical trials

Rosenberger, W. F., & Hu, F. (2004) · Clinical Trials · doi:10.1191/1740774504cn016oa · Cited by 81 · see citing works

Folic acid and prevention of colorectal adenomas: A combined analysis of randomized clinical trials

Figueiredo, J. C., Mott, L. A., Giovannucci, E. L., Wu, K., Cole, B. F., Grainge, M. J., Logan, R. F., & Baron, J. A. (2010) · International Journal of Cancer · doi:10.1002/ijc.25872 · Cited by 78 · see citing works

BCG Revaccination Does Not Protect Against Leprosy in the Brazilian Amazon: A Cluster Randomised Trial

da Cunha, S. S., Alexander, N. D. E., Barreto, M. L., Pereira, E. D. S., Dourado, I. C., Marója, M. D. F., Ichihara, Y., de Brito, S. C., Pereira, S. M., & Rodrigues, L. C. (2008) · PLoS neglected tropical diseases · doi:10.1371/journal.pntd.0000167 · Cited by 65 · see citing works

Absorbent products for incontinence: ‘treatment effects’ and impact on quality of life

Getliffe, K., Fader, M. J., Cottenden, A. M., Jamieson, K., & Green, N. (2007) · Journal of Clinical Nursing · doi:10.1111/j.1365-2702.2007.01812.x · Cited by 59 · see citing works

Clinical Trials of Investigational Antiepileptic Drugs: Monotherapy Designs

Pledger, G. W., & Kramer, L. D. (1991) · Epilepsia · doi:10.1111/j.1528-1157.1991.tb04715.x · Cited by 57 · see citing works

Effect of a Self-management Support Intervention on Asthma Outcomes in Older Adults

Federman, A. D., O’Conor, R. M., Mindlis, I., Hoy‐Rosas, J., Hauser, D., Lurio, J., Shroff, N., Lopez, R., Erblich, J., Wolf, M. S., et al. (2019) · JAMA Internal Medicine · doi:10.1001/jamainternmed.2019.1201 · Cited by 53 · see citing works

The cystic fibrosis therapeutics development network (CF TDN): a paradigm of a clinical trials network for genetic and orphan diseases

Goss, C. H., Mayer-Hamblett, N., Kronmal, R. A., & Ramsey, B. W. (2002) · Advanced Drug Delivery Reviews · doi:10.1016/s0169-409x(02)00163-1 · Cited by 49 · see citing works

Report of a National Institutes of Health–Centers for Disease Control and Prevention workshop on the feasibility of conducting a randomized clinical trial to estimate the long-term health effects of intentional weight loss in obese persons

Yanovski, S. Z., Bain, R. P., & Williamson, D. F. (1999) · American Journal of Clinical Nutrition · doi:10.1093/ajcn/69.3.366 · Cited by 45 · see citing works

Application of machine learning methods in clinical trials for precision medicine

Wang, Y., Carter, B. Z., Li, Z., & Huang, X. (2022) · JAMIA Open · doi:10.1093/jamiaopen/ooab107 · Cited by 36 · see citing works

Statistical issues in interpreting clinical trials

DeMets, D. L. (2004) · Journal of Internal Medicine · doi:10.1111/j.1365-2796.2004.01320.x · Cited by 34 · see citing works

Similarity/Equivalence Trials for Combination Vaccines

Blackwelder, W. C. (1995) · Annals of the New York Academy of Sciences · doi:10.1111/j.1749-6632.1995.tb44465.x · Cited by 33 · see citing works

Allocation of Patients to Conditions in Headache Clinical Trials: Randomization, Stratification, and Treatment Matching

Lipchik, G. L., Nicholson, R. A., & Penzien, D. B. (2005) · Headache The Journal of Head and Face Pain · doi:10.1111/j.1526-4610.2005.05093.x · Cited by 29 · see citing works

Exploring the efficacy of a case management model using DOTS in the adherence of patients with pulmonary tuberculosis

Hsieh, C., Lin, L., Kuo, B. I., Chiang, C., Su, W., & Shih, J.-F. (2007) · Journal of Clinical Nursing · doi:10.1111/j.1365-2702.2006.01924.x · Cited by 28 · see citing works

The Clinical Development of Percutaneous Heart Valve Technology

Vassiliades, T. A., Block, P. C., Cohn, L. H., Adams, D. H., BORER, J. S., Feldman, T. E., Holmes, D. R., Laskey, W. K., Lytle, B. W., Mack, M. J., et al. (2005) · The Annals of Thoracic Surgery · doi:10.1016/j.athoracsur.2005.02.062 · Cited by 28 · see citing works

Maintenance interferon for chronic hepatitis C: More issues than answers?

Everson, G. T. (2000) · Hepatology · doi:10.1053/jhep.2000.16216 · Cited by 26 · see citing works

Use of deception to achieve double-blinding in a clinical trial of Melaleuca alternifolia (tea tree) oil for the treatment of recurrent herpes labialis

Carson, C. F., Smith, D. W., Lampacher, G. J., & Riley, T. V. (2007) · Contemporary Clinical Trials · doi:10.1016/j.cct.2007.04.006 · Cited by 24 · see citing works

The Use of ACE Inhibitors as Renoprotective Agents in Medicaid Patients with Diabetes

Scarsi, K. K., & Bjornson, D. C. (2000) · Annals of Pharmacotherapy · doi:10.1345/aph.19346 · Cited by 20 · see citing works

Strategies for Accessment and Recruitment of Subjects for Nursing Research

Diekmann, J. M., & Smith, J. M. (1989) · Western Journal of Nursing Research · doi:10.1177/019394598901100404 · Cited by 20 · see citing works

Effects of short-term, diet-induced hypercholesterolemia on systemic hemodynamics, myocardial blood flow, and infarct size in awake dogs with acute myocardial infarction.

Sakamoto, S., Kashiki, M., Imai, N., Liang, C., & Hood, W. B. (1991) · Circulation · doi:10.1161/01.cir.84.1.378 · Cited by 17 · see citing works

Validity versus generalizability in clinical trial design and conduct

Hennekens, C. H., & Buring, J. E. (1998) · Journal of Cardiac Failure · doi:10.1016/s1071-9164(98)80014-6 · Cited by 17 · see citing works

Recruitment and Early Retention of Women with Advanced Breast Cancer in a Complementary and Alternative Medicine Trial

Sikorskii, A. E., Wyatt, G. K., Siddiqi, A.-E.-A., & Tamkus, D. M. (2009) · Evidence-based Complementary and Alternative Medicine · doi:10.1093/ecam/nep051 · Cited by 16 · see citing works

Pulmonary response to airway instillation of autologous blood in horses

Derksen, F. J., Williams, K. J., Uhal, B. D., Slocombe, R. F., Feijter-Rupp, H. D., Eberhart, S., BERNEY, C., & ROBINSON, N. E. (2007) · Equine Veterinary Journal · doi:10.2746/042516407x174513 · Cited by 16 · see citing works

Clinical trials

Ratain, J. S., & Hochberg, M. C. (1990) · Arthritis & Rheumatism · doi:10.1002/art.1780330118 · Cited by 16 · see citing works

Dimercaptosuccinic acid for the treatment of hyperhomocysteinemia in hemodialysis patients: A placebo-controlled, double-blind, randomized trial

House, A. A., Eliasziw, M., Urquhart, B. L., Freeman, D. J., & Spence, J. D. (2004) · American Journal of Kidney Diseases · doi:10.1053/j.ajkd.2004.06.010 · Cited by 14 · see citing works

Collaborative asthma self-management evaluation designs

Hindi-Alexander, M. C., Throm, J., & Middleton, E. (1987) · Clinical Reviews in Allergy · doi:10.1007/bf02991198 · Cited by 14 · see citing works

Teaching Courses in Clinical Trial Research Methods

Spilker, B. (1991) · The Journal of Clinical Pharmacology · doi:10.1002/j.1552-4604.1991.tb03727.x · Cited by 13 · see citing works

Regulatory perspectives on clinical trials for trauma, transfusion, and hemostasis

Silverman, T., Aebersold, P. M., Landow, L., & Lindsey, K. (2005) · Transfusion · doi:10.1111/j.0041-1132.2005.00157.x · Cited by 12 · see citing works

Impact of the Patient-Reported Outcomes Management Information System (PROMIS) upon the Design and Operation of Multi-center Clinical Trials: a Qualitative Research Study

Eisenstein, E. L., Diener, L. W., Nahm, M., & Weinfurt, K. P. (2010) · Journal of Medical Systems · doi:10.1007/s10916-010-9429-8 · Cited by 10 · see citing works

Effect of oral citrulline supplementation on whole body protein metabolism in adult patients with short bowel syndrome: A pilot, randomized, double-blind, cross-over study

Jirka, A., Layec, S., Picot, D., Bernon-Ferreira, S., Grasset, N., Flet, L., Thibault, R., & Darmaun, D. (2019) · Clinical Nutrition · doi:10.1016/j.clnu.2018.12.030 · Cited by 10 · see citing works

Drug discovery and development

Rang, H. P., Dale, M. M., Ritter, J. M., Flower, R. J., & Henderson, G. (2012) · Elsevier eBooks · doi:10.1016/b978-0-7020-3471-8.00060-3 · Cited by 10 · see citing works

Assessing Treatment Results for Benign Prostatic Hyperplasia

Wein, A. J. (1993) · Springer eBooks · doi:10.1007/978-3-642-45723-4_4 · Cited by 9 · see citing works

ASSESSING TREATMENT RESULTS IN BENIGN PROSTATIC HYPERPLASIA

Wein, A. J. (1995) · Urologic Clinics of North America · doi:10.1016/s0094-0143(21)00670-4 · Cited by 9 · see citing works

When Practices, Promises, Profits, and Policies Outpace Hard Evidence: The Post‐Menopausal Hormone Debate

Naughton, M. J., Jones, A. S., & Shumaker, S. A. (2005) · Journal of Social Issues · doi:10.1111/j.0022-4537.2005.00399.x · Cited by 8 · see citing works

Review: clinical trials in peptic ulcer disease—problems of methodology and interpretation

Lauritsen, K., & Rask-Madsen, J. (1987) · Alimentary Pharmacology & Therapeutics · doi:10.1111/j.1365-2036.1987.tb00610.x · Cited by 8 · see citing works

Therapeutic apheresis in neurological disorders

Weinstein, R. S. (2000) · Journal of Clinical Apheresis · doi:10.1002/(sici)1098-1101(2000)15:1/2 · Cited by 78 · see citing works

Conducting social experiments

Boruch, R. F. (1987) · New Directions for Program Evaluation · doi:10.1002/ev.1452 · Cited by 6 · see citing works

Basic Design Considerations for Clinical Trials in Oncology

Piantadosi, S., Saijo, N., & Tamura, T. (1992) · Japanese Journal of Cancer Research · doi:10.1111/j.1349-7006.1992.tb00124.x · Cited by 5 · see citing works

Statistical practice and statistical education in cardiology.

Ware, J. H. (1987) · Circulation · doi:10.1161/01.cir.75.2.307 · Cited by 5 · see citing works

Herz-Kreislauf-Prävention

Hüllemann, K.-D. (1986) · Springer eBooks · doi:10.1007/978-3-642-70910-4_2 · Cited by 5 · see citing works

TrialEnroll: Predicting Clinical Trial Enrollment Success with Deep & Cross Network and Large Language Models

Yue, L., Xing, S., Chen, J., & Fu, T. (2024) · ACM International Conference on Bioinformatics, Computational Biology, and Health Informatics (ACM-BCB) · doi:10.1145/3698587.3701375 · Cited by 4 · see citing works

A survey of physician efficacy requirements to plan clinical trials

Oremus, M., Collet, J., Corcos, J., & Shapiro, S. H. (2002) · Pharmacoepidemiology and Drug Safety · doi:10.1002/pds.750 · Cited by 4 · see citing works

Statistical methods in experimentation recommendation models for discovering gene regulation pathways

Yoo, C. (2013) · Wiley Interdisciplinary Reviews Computational Statistics · doi:10.1002/wics.1244 · Cited by 1 · see citing works

Design of clinical trials

Gordis, L. (1986) · Journal of Allergy and Clinical Immunology · doi:10.1016/0091-6749(86)90007-2 · Cited by 1 · see citing works

The Design of Interventional Trials in Transfusion Medicine

Hébert, P. C., Tinmouth, A. T., & Fergusson, D. A. (2005) · doi:10.1002/9780470988411.ch37

Purpose and Design of Clinical Trials

Warrington, S. (2009) · doi:10.1002/9781444317800.ch6

ModernIn VivoEvaluations and Clinical Trials

Saad, B., & Said, O. (2011) · doi:10.1002/9780470944363.ch14

Methodologie klinischer Forschungsregister

Mau, J., Axmann, D., Bluhmki, E., & Scheytt, C. (1988) · Medizinische Informatik und Statistik · doi:10.1007/978-3-642-83520-9_9

Clinical trials in chronic neuromuscular diseases: Proceedings of a colloquium at Totts Gap Institute, Bangor, PA, sponsored by The Muscular Dystrophy Association

Wolf, S., & Lewis, N. J. (1985) · Muscle & Nerve · doi:10.1002/mus.880080602

Evaluating causality in the aids epidemic: The role of mathematical models

Kaldor, J. M. (1995) · Environmetrics · doi:10.1002/env.3170060519

Book Reviews

Farrell, R. (1987) · Journal of the American Statistical Association · doi:10.1080/01621459.1987.10478437

Reply

Piccini, J. P., & Kong, D. F. (2010) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2009.12.011

Bibliography

Good, P. I., & Hardin, J. W. (2012) · Wiley eBooks · doi:10.1002/9781118360125.biblio

References

(2013) · Wiley series in probability and statistics · doi:10.1002/9781118458167.refs

References

Pocock, S. (2013) · Clinical Trials · doi:10.1002/9781118793916.refs

Bibliography

Good, P. I., & Hardin, J. W. (2008) · doi:10.1002/9780470473924.biblio

Alpha‐Spending Function

DeMets, D. L., & Lan, K. K. G. (2005) · Encyclopedia of Statistical Sciences · doi:10.1002/0471667196.ess7286

Book Reviews

(2012) · Journal of the American Statistical Association · doi:10.1080/01621459.2012.664485

Book Reviews

(1995) · Journal of the American Statistical Association · doi:10.1080/01621459.1995.10476524

The Efficacy of Lactobacillus ferment Lysate-Derived Prebiotics in Mist and Serum Form on Skin Inflammation: A Two-Week Prospective Study

Wong, V., Abdulbaky, A., Havard, C. M., Chikwendu, C., & Redelbach-Gaisford, M. (2025) · Journal of Dermatology Research · doi:10.46889/jdr.2025.6316

Methodology of Clinical Drug Trials.

North, P. M., Spriet, A., & Simon, P. (1986) · Biometrics · doi:10.2307/2531226 · Cited by 1 · see citing works

An Introduction to Clinical Trials

Villar, S. S. (2026) · Biometrics · doi:10.1093/biomtc/ujag007

Fundamentals of planning a clinical trial. II.

Ozick, J. (1967) · PubMed

Fundamentals of Clinical Trials. (2nd ed.).

George, S. L., Friedman, L. M., Furberg, C. D., & DeMets, D. L. (1987) · Journal of the American Statistical Association · doi:10.2307/2289197 · Cited by 124 · see citing works

Preface: Recent advances in clinical trial methodology

正彦, 五. (2022) · Japanese Journal of Biometrics · doi:10.5691/jjb.43.2