A flexible design for advanced Phase I/II clinical trials with continuous efficacy endpoints

Mozgunov, P. & Jaki, T., 1/11/2019, In: Biometrical Journal. 61, 6, p. 1477-1492 16 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Subgroup analysis of treatment effects for misclassified biomarkers with time-to-event data

Wan, F., Titman, A. & Jaki, T., 1/11/2019, In: Journal of the Royal Statistical Society: Series C (Applied Statistics). 68, 5, p. 1447-1463 17 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis

Schanberg, L. E., Ramanan, A. V., De Benedetti, F., Beukelman, T., Eakin, G. S., Del Gaizo, V., Ringold, S., Vesely, R., Schrandt, S., Jaki, T., Bili, A., Chung, J. B., De Bono, S., Douglass, W., Enejosa, J. V., Kanik, K. S., Knobe, K., Kunder, R., Leite-Schnell, J. C., Suehiro, R. M., & 6 othersWong, R. L., Mieszkalski, K. L., Marrow, L. C., Siebenaler, K., Fraulo, E. & Kimura, Y., 1/12/2019, In: Arthritis and Rheumatology. 71, 12, p. 1976-1984 9 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A quantitative framework to inform extrapolation decisions in children

Wadsworth, I., Hampson, L. V., Jaki, T., Sills, G. J., Marson, A. G. & Appleton, R., 1/02/2020, In: Journal of the Royal Statistical Society: Series A Statistics in Society. 183, 2, p. 515-534 20 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A randomised controlled trial of rosuvastatin for the prevention of aminoglycoside-induced kidney toxicity in children with cystic fibrosis

McWilliam, S. J., Rosala-Hallas, A., Jones, A. P., Shaw, V., Greenhalf, W., Jaki, T., Smyth, A. R., Smyth, R. L. & Pirmohamed, M., 4/02/2020, In: Scientific Reports. 10, 1, 9 p., 1796.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Improving safety of the continual reassessment method via a modified allocation rule

Mozgunov, P. & Jaki, T., 30/03/2020, In: Statistics in Medicine. 39, 7, p. 906-922 17 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A benchmark for dose finding studies with continuous outcomes

Mozgunov, P., Jaki, T. F. & Paoletti, X., 1/04/2020, In: Biostatistics. 21, 2, p. 189–201 13 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Designing and evaluating dose-escalation studies made easy: The MoDEsT web app

Pallmann, P., Wan, F., Mander, A. P., Wheeler, G. M., Yap, C., Clive, S., Hampson, L. V. & Jaki, T., 1/04/2020, In: Clinical Trials. 17, 2, p. 147-156 10 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Repeated measures regression mixture models

Kim, M., Van Horn, M. L., Jaki, T., Vermunt, J., Feaster, D., Lichstein, K. L., Taylor, D. J., Riedel, B. W. & Bush, A. J., 1/04/2020, In: Behavior Research Methods. 52, 2, p. 591-606 16 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Creating a framework for conducting randomized clinical trials during disease outbreaks

Dean, N. E., Gsell, P-S., Brookmeyer, R., Crawford, F. W., Donnelly, C. A., Ellenberg, S. S., Fleming, T. R., Halloran, E. M., Horby, P., Jaki, T., Krause, P. R., Longini, I. M., Mulangu, S., Muyembe-Tamfum, J-J., Nason, M. C., Smith, P. G., Wang, R., Henao-Restrepo, A. M. & De Gruttola, V., 2/04/2020, In: New England Journal of Medicine. 382, 14, p. 1366-1369 4 p.

Research output: Contribution to Journal/Magazine › Editorial › peer-review

A comparison of stochastic programming methods for portfolio level decision-making

Graham, E., Jaki, T. & Harbron, C., 1/05/2020, In: Journal of Biopharmaceutical Statistics. 30, 3, p. 405-429 25 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

TAILoR (TelmisArtan and InsuLin Resistance in Human Immunodeficiency Virus [HIV]): An Adaptive-design, Dose-ranging Phase IIb Randomized Trial of Telmisartan for the Reduction of Insulin Resistance in HIV-positive Individuals on Combination Antiretroviral Therapy

Pushpakom, S., Kolamunnage-Dona, R., Taylor, C., Foster, T., Spowart, C., García-Fiñana, M., Kemp, G. J., Jaki, T., Khoo, S., Williamson, P. & Pirmohamed, M., 15/05/2020, In: Clinical infectious diseases : an official publication of the Infectious Diseases Society of America. 70, 10, p. 2062-2072 11 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Remdesivir in adults with severe COVID-19: a randomised, double-blind, placebo-controlled, multicentre trial

Wang, Y., Zhang, D., Du, G., Du, R., Zhao, J., Jin, Y., Fu, S., Gao, L., Cheng, Z., Lu, Q., Hu, Y., Luo, G., Wang, K., Lu, Y., Li, H., Wang, S., Ruan, S., Yang, C., Mei, C., Ding, D., & 25 othersWu, F., Tang, X., Ye, X., Ye, Y., Liu, B., Yang, J., Yin, W., Wang, A., Fan, G., Zhou, F., Liu, Z., Gu, X., Xu, J., Shang, L., Zhang, Y., Cao, L., Guo, T., Wan, Y., Qin, H., Jiang, Y., Jaki, T., Hayden, F. G., Horby, P. W., Cao, B. & Wang, C., 16/05/2020, In: The Lancet. p. 1569-1578 10 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Estimation of treatment effects following a sequential trial of multiple treatments

Whitehead, J., Desai, Y. & Jaki, T., 20/05/2020, In: Statistics in Medicine. 39, 11, p. 1593-1609 17 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Evaluation of the efficacy and safety of intravenous remdesivir in adult patients with severe COVID-19: Study protocol for a phase 3 randomized, double-blind, placebo-controlled, multicentre trial

Wang, Y., Zhou, F., Zhang, D., Zhao, J., Du, R., Hu, Y., Cheng, Z., Gao, L., Jin, Y., Luo, G., Fu, S., Lu, Q., Du, G., Wang, K., Lu, Y., Fan, G., Zhang, Y., Liu, Y., Ruan, S., Liu, W., & 5 othersJaki, T., Hayden, F. G., Horby, P. W., Cao, B. & Wang, C., 24/05/2020, In: Trials. 21, 1, 11 p., 422.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

The adaptive designs CONSORT extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design

Dimairo, M., Pallmann, P., Wason, J., Todd, S., Jaki, T., Julious, S. A., Mander, A. P., Weir, C. J., Koenig, F., Walton, M. K., Nicholl, J. P., Coates, E., Biggs, K., Hamasaki, T., Proschan, M. A., Scott, J. A., Ando, Y., Hind, D., Altman, D. G. & Group, ACE. C., 17/06/2020, In: Trials. 21, 1, 34 p., 528.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

The Adaptive designs CONSORT Extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design

Dimairo, M., Pallmann, P., Wason, J., Todd, S., Jaki, T., Julious, S. A., Mander, A. P., Weir, C. J., Koenig, F., Walton, M. K., Nicholl, J. P., Coates, E., Biggs, K., Hamasaki, T., Proschan, M. A., Scott, J. A., Ando, Y., Hind, D., Altman, D. G. & Group, ACE. C., 17/06/2020, In: BMJ. 369, 31 p., m115.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

AGILE-ACCORD: A Randomized, Multicentre, Seamless, Adaptive Phase I/II Platform Study to Determine the Optimal Dose, Safety and Efficacy of Multiple Candidate Agents for the Treatment of COVID-19: A structured summary of a study protocol for a randomised platform trial

UK NIHR Community, 19/06/2020, In: Trials. 21, 1, 3 p., 544.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A Critical Review of Graphics for Subgroup Analyses in Clinical Trials

Ballarini, N. M., Chiu, Y-D., Koenig, F., Posch, M. & Jaki, T., 1/09/2020, In: Pharmaceutical Statistics. 19, 5, p. 541-560 20 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Exposure–response modelling approaches for determining optimal dosing rules in children

Wadsworth, I., Hampson, L. V., Bornkamp, B. & Jaki, T., 1/09/2020, In: Statistical Methods in Medical Research. 29, 9, p. 2583-2602 20 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A comparison of phase I dose-finding designs in clinical trials with monotonicity assumption violation

Abbas, R., Rossoni, C., Jaki, T., Paoletti, X. & Mozgunov, P., 1/10/2020, In: Clinical Trials. 17, 5, p. 522-534 13 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A surface-free design for phase I dual-agent combination trials

Mozgunov, P., Gasparini, M. & Jaki, T., 1/10/2020, In: Statistical Methods in Medical Research. 29, 10, p. 3093-3109 17 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Efficient Adaptive Designs for Clinical Trials of Interventions for COVID-19

Stallard, N., Hampson, L., Benda, N., Brannath, W., Burnett, T., Friede, T., Kimani, P. K., Koenig, F., Krisam, J., Mozgunov, P., Posch, M., Wason, J., Wassmer, G., Whitehead, J., Williamson, S. F., Zohar, S. & Jaki, T., 1/10/2020, In: Statistics in Biopharmaceutical Research. 12, 4, p. 483-497 15 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Endpoints for randomized controlled clinical trials for COVID-19 treatments

Dodd, L. E., Follmann, D., Wang, J., Koenig, F., Korn, L. L., Schoergenhofer, C., Proschan, M., Hunsberger, S., Bonnett, T., Makowski, M., Belhadi, D., Wang, Y., Cao, B., Mentre, F. & Jaki, T., 1/10/2020, In: Clinical Trials. 17, 5, p. 472-482 11 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Using a Dose-Finding Benchmark to Quantify the Loss Incurred by Dichotomisation in Phase II Dose-Ranging Studies

Mozgunov, P., Jaki, T. & Paoletti, X., 1/11/2020, In: Biometrical Journal. 62, 7, p. 1717-1729 13 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Using a dose-finding benchmark to quantify the loss incurred by dichotomization in Phase II dose-ranging studies

Mozgunov, P., Jaki, T. & Paoletti, X., 1/11/2020, In: Biometrical Journal. 62, 7, p. 1717-1729 13 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

An information-theoretic approach for selecting arms in clinical trials

Mozgunov, P. & Jaki, T. F., 10/11/2020, In: Journal of the Royal Statistical Society: Series B (Statistical Methodology). 82, 5, p. 1223-1247 25 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs

Burnett, T., Mozgunov, P., Pallmann, P., Villar, S. S., Wheeler, G. M. & Jaki, T., 19/11/2020, In: BMC Medicine. 18, 1, 21 p., 352.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Methods for Non-Compartmental Pharmacokinetic Analysis With Observations Below the Limit of Quantification

Barnett, H. Y., Geys, H., Jacobs, T. & Jaki, T., 2/01/2021, In: Statistics in Biopharmaceutical Research. 13, 1, p. 59-70 12 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Using an Interaction Parameter in Model-Based Phase I Trials for Combination Treatments? A Simulation Study

Mozgunov, P., Knight, R., Barnett, H. & Jaki, T., 5/01/2021, In: International Journal of Environmental Research and Public Health. 18, 1, 19 p., 345.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Recurrent events modelling of haemophilia bleeding events

Titman, A., Wolfsegger, M. J. & Jaki, T., 1/03/2021, In: Journal of the Royal Statistical Society: Series C (Applied Statistics). 70, 2, p. 351-371 21 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Statistical consideration when adding new arms to ongoing clinical trials: the potentials and the caveats

Lee, K. M., Brown, L. C., Jaki, T., Stallard, N. & Wason, J., 10/03/2021, In: Trials. 22, 1, 10 p., 203.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Assessing goodness-of-fit for evaluation of dose-proportionality

Wolfsegger, M. J., Bauer, A., Labes, D., Schütz, H., Vonk, R., Lang, B., Lehr, S., Jaki, T. F., Engl, W. & Hale, M. D., 31/03/2021, In: Pharmaceutical Statistics. 20, 2, p. 272-281 10 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Bridging across patient subgroups in phase I oncology trials that incorporate animal data

Zheng, H., Hampson, L. V. & Jaki, T., 1/04/2021, In: Statistical Methods in Medical Research. 30, 4, p. 1057-1071 15 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Applying methods for personalized medicine to the treatment of alcohol use disorder

Kuhlemeier, A., Desai, Y., Tonigan, A., Witkiewitz, K., Jaki, T., Hsiao, Y-Y., Chang, C. & Van Horn, M. L., 30/04/2021, In: Journal of Consulting and Clinical Psychology. 89, 4, p. 288-300 13 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Optimizing subgroup selection in two-stage adaptive enrichment and umbrella designs

Ballarini, N. M., Burnett, T., Jaki, T., Jennison, C., König, F. & Posch, M., 31/05/2021, In: Statistics in Medicine. 40, 12, p. 2939-2956 18 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Study to evaluate the optimal dose of remifentanil required to ensure apnea during magnetic resonance imaging of the heart under general anesthesia

Arnold, P., Sanaulla, S., Hampson, L. V., Davis, A., Tan, J., Cowen, R., Kaleem, M., Williams, A., Wadsworth, I. & Jaki, T., 31/05/2021, In: Paediatric Anesthesia. 31, 5, p. 548-556 9 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

AGILE: a seamless phase I/IIa platform for the rapid evaluation of candidates for COVID-19 treatment: an update to the structured summary of a study protocol for a randomised platform trial letter

Griffiths, G. O., FitzGerald, R., Jaki, T., Corkhill, A., Reynolds, H., Ewings, S., Condie, S., Tilt, E., Johnson, L., Radford, M., Simpson, C., Saunders, G., Yeats, S., Mozgunov, P., Tansley-Hancock, O., Martin, K., Downs, N., Eberhart, I., Martin, J. W. B., Goncalves, C., & 18 othersSong, A., Fletcher, T., Byrne, K., Lalloo, D. G., Owen, A., Jacobs, M., Walker, L., Lyon, R., Woods, C., Gibney, J., Chiong, J., Chandiwana, N., Jacob, S., Lamorde, M., Orrell, C., Pirmohamed, M., Khoo, S. & investigators, O. B. O. T. AGILE., 26/07/2021, In: Trials. 22, 1, 5 p., 487.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Proposing a Core Outcome Set for Physical Activity and Exercise Interventions in People With Rare Neurological Conditions

Ramdharry, G., Buscemi, V., Boaz, A., Dawes, H., Jaki, T., Jones, F., Marsden, J., Paul, L., Playle, R., Randell, E., Robling, M., Rochester, L. & Busse, M., 21/10/2021, (E-pub ahead of print) In: Frontiers in Rehabilitation Sciences. 2, 13 p., 705474.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Costs and staffing resource requirements for adaptive clinical trials: quantitative and qualitative results from the Costing Adaptive Trials project

Wilson, N., Biggs, K., Bowden, S., Brown, J., Dimairo, M., Flight, L., Hall, J., Hockaday, A., Jaki, T., Lowe, R., Murphy, C., Pallmann, P., Pilling, M. A., Snowdon, C., Sydes, M. R., Villar, S. S., Weir, C. J., Welburn, J., Yap, C., Maier, R., & 2 othersHancock, H. & Wason, J. M. S., 26/10/2021, In: BMC Medicine. 19, 1, 17 p., 251.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A permutation test for assessing the presence of individual differences in treatment effects

Chang, C., Jaki, T., Sadiq, M. S., Kuhlemeier, A., Feaster, D., Cole, N., Lamont, A., Oberski, D., Desai, Y. & Lee Van Horn, M., 1/11/2021, In: Statistical Methods in Medical Research. 30, 11, p. 2369-2381 13 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Optimal dose and safety of molnupiravir in patients with early SARS-CoV-2: a Phase I, open-label, dose-escalating, randomized controlled study

Khoo, S. H., Fitzgerald, R., Fletcher, T., Ewings, S., Jaki, T., Lyon, R., Downs, N., Walker, L., Tansley-Hancock, O., Greenhalf, W., Woods, C., Reynolds, H., Marwood, E., Mozgunov, P., Adams, E., Bullock, K., Holman, W., Bula, M. D., Gibney, J. L., Saunders, G., & 18 othersCorkhill, A., Hale, C., Thorne, K., Chiong, J., Condie, S., Pertinez, H., Painter, W., Wrixon, E., Johnson, L., Yeats, S., Mallard, K., Radford, M., Fines, K., Shaw, V., Owen, A., Lalloo, D. G., Jacobs, M. & Griffiths, G., 12/11/2021, In: Journal of Antimicrobial Chemotherapy. 76, 12, p. 3286-3295 10 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Practical recommendations for implementing a Bayesian adaptive phase I design during a pandemic

Ewings, S., Saunders, G., Jaki, T. & Mozgunov, P., 20/01/2022, In: BMC Medical Research Methodology. 22, 1, 15 p., 25.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

The need for reporting guidelines for early phase dose-finding trials: Dose-Finding CONSORT Extension

Yap, C., Bedding, A., de Bono, J., Dimairo, M., Espinasse, A., Evans, J., Hopewell, S., Jaki, T., Kightley, A., Lee, S., Liu, R., Mander, A., Solovyeva, O. & Weir, C. J., 31/01/2022, In: Nature Medicine. 28, 1, p. 6-7 2 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A dose-finding design for dual-agent trials with patient-specific doses for one agent with application to an opiate detoxification trial

Mozgunov, P., Cro, S., Lingford-Hughes, A., Paterson, L. M. & Jaki, T., 31/03/2022, In: Pharmaceutical Statistics. 21, 2, p. 476-495 20 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

An order restricted multi-arm multi-stage clinical trial design

Serra, A., Mozgunov, P. & Jaki, T., 30/04/2022, In: Statistics in Medicine. 41, 9, p. 1613-1626 14 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Individual differences in the effects of the ACTION-PAC intervention: an application of personalized medicine in the prevention and treatment of obesity

Kuhlemeier, A., Jaki, T., Jimenez, E. Y., Kong, A. S., Gill, H., Chang, C., Resnicow, K., Wilson, D. K. & Van Horn, M. L., 30/04/2022, In: Journal of Behavioral Medicine. 45, 2, p. 211-226 16 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

A Benchmark for Dose Finding Studies with Unknown Ordering

Mozgunov, P., Paoletti, X. & Jaki, T., 31/07/2022, In: Biostatistics. 23, 3, p. 721-737 17 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

What is the expected benefit of patient-centric clinical development in oncology?

Serra, A., Mozgunov, P., Jaki, T. & Rigat, F., 31/07/2022, In: Journal of Biopharmaceutical Statistics. 32, 3, p. 414-426 13 p.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Practical guidance for planning resources required to support publicly-funded adaptive clinical trials

Wason, J. M. S., Dimairo, M., Biggs, K., Bowden, S., Brown, J., Flight, L., Hall, J., Jaki, T., Lowe, R., Pallmann, P., Pilling, M. A., Snowdon, C., Sydes, M. R., Villar, S. S., Weir, C. J., Wilson, N., Yap, C., Hancock, H. & Maier, R., 10/08/2022, In: BMC Medicine. 20, 1, 12 p., 254.

Research output: Contribution to Journal/Magazine › Journal article › peer-review