Trial design and objectives for castration-resistant prostate cancer: Updated recommendations from the prostate cancer clinical trials working group 3

Howard I. Scher, Michael J. Morris, Walter M. Stadler, Celestia Higano, Ethan Basch, Karim Fizazi, Emmanuel S. Antonarakis, Tomasz (Tom) Beer, Michael A. Carducci, Kim N. Chi, Paul G. Corn, Johann S. De Bono, Robert Dreicer, Daniel J. George, Elisabeth I. Heath, Maha Hussain, Wm Kevin Kelly, Glenn Liu, Christopher Logothetis, David NanusMark N. Stein, Dana E. Rathkopf, Susan F. Slovin, Charles J. Ryan, Oliver Sartor, Eric J. Small, Matthew Raymond Smith, Cora N. Sternberg, Mary Ellen Taplin, George Wilding, Peter S. Nelson, Lawrence H. Schwartz, Susan Halabi, Philip W. Kantoff, Andrew J. Armstrong

    Research output: Contribution to journalArticle

    337 Citations (Scopus)

    Abstract

    Purpose Evolving treatments, disease phenotypes, and biology, together with a changing drug development environment, have created the need to revise castration-resistant prostate cancer (CRPC) clinical trial recommendations to succeed those from prior Prostate Cancer Clinical Trials Working Groups. Methods An international expert committee of prostate cancer clinical investigators (the Prostate Cancer Clinical Trials Working Group 3 [PCWG3]) was reconvened and expanded and met in 2012-2015 to formulate updated criteria on the basis of emerging trial data and validation studies of the Prostate Cancer Clinical Trials Working Group 2 recommendations. Results PCWG3 recommends that baseline patient assessment include tumor histology, detailed records of prior systemic treatments and responses, and a detailed reporting of disease subtypes based on an anatomic pattern of metastatic spread. New recommendations for trial outcome measures include the time to event end point of symptomatic skeletal events, as well as time to first metastasis and time to progression for trials in the nonmetastatic CRPC state. PCWG3 introduces the concept of no longer clinically benefiting to underscore the distinction between first evidence of progression and the clinical need to terminate or change treatment, and the importance of documenting progression in existing lesions as distinct from the development of new lesions. Serial biologic profiling using tumor samples from biopsies, blood-based diagnostics, and/or imaging is also recommended to gain insight into mechanisms of resistance and to identify predictive biomarkers of sensitivity for use in prospective trials. Conclusion PCWG3 moves drug development closer to unmet needs in clinical practice by focusing on disease manifestations most likely to affect prognosis adversely for therapeutics tested in both nonmetastatic and metastatic CRPC populations. Consultation with regulatory authorities is recommended if a trial is intended to seek support for drug approval.

    Original languageEnglish (US)
    Pages (from-to)1402-1418
    Number of pages17
    JournalJournal of Clinical Oncology
    Volume34
    Issue number12
    DOIs
    StatePublished - Apr 20 2016

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    Castration
    Prostatic Neoplasms
    Clinical Trials
    Drug Approval
    Validation Studies
    Diagnostic Imaging
    Therapeutics
    Pharmaceutical Preparations
    Neoplasms
    Histology
    Referral and Consultation
    Biomarkers
    Research Personnel
    Outcome Assessment (Health Care)
    Neoplasm Metastasis
    Phenotype
    Biopsy

    ASJC Scopus subject areas

    • Medicine(all)
    • Oncology
    • Cancer Research

    Cite this

    Trial design and objectives for castration-resistant prostate cancer : Updated recommendations from the prostate cancer clinical trials working group 3. / Scher, Howard I.; Morris, Michael J.; Stadler, Walter M.; Higano, Celestia; Basch, Ethan; Fizazi, Karim; Antonarakis, Emmanuel S.; Beer, Tomasz (Tom); Carducci, Michael A.; Chi, Kim N.; Corn, Paul G.; De Bono, Johann S.; Dreicer, Robert; George, Daniel J.; Heath, Elisabeth I.; Hussain, Maha; Kelly, Wm Kevin; Liu, Glenn; Logothetis, Christopher; Nanus, David; Stein, Mark N.; Rathkopf, Dana E.; Slovin, Susan F.; Ryan, Charles J.; Sartor, Oliver; Small, Eric J.; Smith, Matthew Raymond; Sternberg, Cora N.; Taplin, Mary Ellen; Wilding, George; Nelson, Peter S.; Schwartz, Lawrence H.; Halabi, Susan; Kantoff, Philip W.; Armstrong, Andrew J.

    In: Journal of Clinical Oncology, Vol. 34, No. 12, 20.04.2016, p. 1402-1418.

    Research output: Contribution to journalArticle

    Scher, HI, Morris, MJ, Stadler, WM, Higano, C, Basch, E, Fizazi, K, Antonarakis, ES, Beer, TT, Carducci, MA, Chi, KN, Corn, PG, De Bono, JS, Dreicer, R, George, DJ, Heath, EI, Hussain, M, Kelly, WK, Liu, G, Logothetis, C, Nanus, D, Stein, MN, Rathkopf, DE, Slovin, SF, Ryan, CJ, Sartor, O, Small, EJ, Smith, MR, Sternberg, CN, Taplin, ME, Wilding, G, Nelson, PS, Schwartz, LH, Halabi, S, Kantoff, PW & Armstrong, AJ 2016, 'Trial design and objectives for castration-resistant prostate cancer: Updated recommendations from the prostate cancer clinical trials working group 3', Journal of Clinical Oncology, vol. 34, no. 12, pp. 1402-1418. https://doi.org/10.1200/JCO.2015.64.2702
    Scher, Howard I. ; Morris, Michael J. ; Stadler, Walter M. ; Higano, Celestia ; Basch, Ethan ; Fizazi, Karim ; Antonarakis, Emmanuel S. ; Beer, Tomasz (Tom) ; Carducci, Michael A. ; Chi, Kim N. ; Corn, Paul G. ; De Bono, Johann S. ; Dreicer, Robert ; George, Daniel J. ; Heath, Elisabeth I. ; Hussain, Maha ; Kelly, Wm Kevin ; Liu, Glenn ; Logothetis, Christopher ; Nanus, David ; Stein, Mark N. ; Rathkopf, Dana E. ; Slovin, Susan F. ; Ryan, Charles J. ; Sartor, Oliver ; Small, Eric J. ; Smith, Matthew Raymond ; Sternberg, Cora N. ; Taplin, Mary Ellen ; Wilding, George ; Nelson, Peter S. ; Schwartz, Lawrence H. ; Halabi, Susan ; Kantoff, Philip W. ; Armstrong, Andrew J. / Trial design and objectives for castration-resistant prostate cancer : Updated recommendations from the prostate cancer clinical trials working group 3. In: Journal of Clinical Oncology. 2016 ; Vol. 34, No. 12. pp. 1402-1418.
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    abstract = "Purpose Evolving treatments, disease phenotypes, and biology, together with a changing drug development environment, have created the need to revise castration-resistant prostate cancer (CRPC) clinical trial recommendations to succeed those from prior Prostate Cancer Clinical Trials Working Groups. Methods An international expert committee of prostate cancer clinical investigators (the Prostate Cancer Clinical Trials Working Group 3 [PCWG3]) was reconvened and expanded and met in 2012-2015 to formulate updated criteria on the basis of emerging trial data and validation studies of the Prostate Cancer Clinical Trials Working Group 2 recommendations. Results PCWG3 recommends that baseline patient assessment include tumor histology, detailed records of prior systemic treatments and responses, and a detailed reporting of disease subtypes based on an anatomic pattern of metastatic spread. New recommendations for trial outcome measures include the time to event end point of symptomatic skeletal events, as well as time to first metastasis and time to progression for trials in the nonmetastatic CRPC state. PCWG3 introduces the concept of no longer clinically benefiting to underscore the distinction between first evidence of progression and the clinical need to terminate or change treatment, and the importance of documenting progression in existing lesions as distinct from the development of new lesions. Serial biologic profiling using tumor samples from biopsies, blood-based diagnostics, and/or imaging is also recommended to gain insight into mechanisms of resistance and to identify predictive biomarkers of sensitivity for use in prospective trials. Conclusion PCWG3 moves drug development closer to unmet needs in clinical practice by focusing on disease manifestations most likely to affect prognosis adversely for therapeutics tested in both nonmetastatic and metastatic CRPC populations. Consultation with regulatory authorities is recommended if a trial is intended to seek support for drug approval.",
    author = "Scher, {Howard I.} and Morris, {Michael J.} and Stadler, {Walter M.} and Celestia Higano and Ethan Basch and Karim Fizazi and Antonarakis, {Emmanuel S.} and Beer, {Tomasz (Tom)} and Carducci, {Michael A.} and Chi, {Kim N.} and Corn, {Paul G.} and {De Bono}, {Johann S.} and Robert Dreicer and George, {Daniel J.} and Heath, {Elisabeth I.} and Maha Hussain and Kelly, {Wm Kevin} and Glenn Liu and Christopher Logothetis and David Nanus and Stein, {Mark N.} and Rathkopf, {Dana E.} and Slovin, {Susan F.} and Ryan, {Charles J.} and Oliver Sartor and Small, {Eric J.} and Smith, {Matthew Raymond} and Sternberg, {Cora N.} and Taplin, {Mary Ellen} and George Wilding and Nelson, {Peter S.} and Schwartz, {Lawrence H.} and Susan Halabi and Kantoff, {Philip W.} and Armstrong, {Andrew J.}",
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    T1 - Trial design and objectives for castration-resistant prostate cancer

    T2 - Updated recommendations from the prostate cancer clinical trials working group 3

    AU - Scher, Howard I.

    AU - Morris, Michael J.

    AU - Stadler, Walter M.

    AU - Higano, Celestia

    AU - Basch, Ethan

    AU - Fizazi, Karim

    AU - Antonarakis, Emmanuel S.

    AU - Beer, Tomasz (Tom)

    AU - Carducci, Michael A.

    AU - Chi, Kim N.

    AU - Corn, Paul G.

    AU - De Bono, Johann S.

    AU - Dreicer, Robert

    AU - George, Daniel J.

    AU - Heath, Elisabeth I.

    AU - Hussain, Maha

    AU - Kelly, Wm Kevin

    AU - Liu, Glenn

    AU - Logothetis, Christopher

    AU - Nanus, David

    AU - Stein, Mark N.

    AU - Rathkopf, Dana E.

    AU - Slovin, Susan F.

    AU - Ryan, Charles J.

    AU - Sartor, Oliver

    AU - Small, Eric J.

    AU - Smith, Matthew Raymond

    AU - Sternberg, Cora N.

    AU - Taplin, Mary Ellen

    AU - Wilding, George

    AU - Nelson, Peter S.

    AU - Schwartz, Lawrence H.

    AU - Halabi, Susan

    AU - Kantoff, Philip W.

    AU - Armstrong, Andrew J.

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    N2 - Purpose Evolving treatments, disease phenotypes, and biology, together with a changing drug development environment, have created the need to revise castration-resistant prostate cancer (CRPC) clinical trial recommendations to succeed those from prior Prostate Cancer Clinical Trials Working Groups. Methods An international expert committee of prostate cancer clinical investigators (the Prostate Cancer Clinical Trials Working Group 3 [PCWG3]) was reconvened and expanded and met in 2012-2015 to formulate updated criteria on the basis of emerging trial data and validation studies of the Prostate Cancer Clinical Trials Working Group 2 recommendations. Results PCWG3 recommends that baseline patient assessment include tumor histology, detailed records of prior systemic treatments and responses, and a detailed reporting of disease subtypes based on an anatomic pattern of metastatic spread. New recommendations for trial outcome measures include the time to event end point of symptomatic skeletal events, as well as time to first metastasis and time to progression for trials in the nonmetastatic CRPC state. PCWG3 introduces the concept of no longer clinically benefiting to underscore the distinction between first evidence of progression and the clinical need to terminate or change treatment, and the importance of documenting progression in existing lesions as distinct from the development of new lesions. Serial biologic profiling using tumor samples from biopsies, blood-based diagnostics, and/or imaging is also recommended to gain insight into mechanisms of resistance and to identify predictive biomarkers of sensitivity for use in prospective trials. Conclusion PCWG3 moves drug development closer to unmet needs in clinical practice by focusing on disease manifestations most likely to affect prognosis adversely for therapeutics tested in both nonmetastatic and metastatic CRPC populations. Consultation with regulatory authorities is recommended if a trial is intended to seek support for drug approval.

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