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Can RECIST response predict success in phase 3 trials in men with metastatic castration-resistant prostate cancer?

Prostate Cancer and Prostatic Diseases volume 21, pages 419–430 (2018)Cite this article

Subjects

Abstract

Background

Intermediate endpoints are needed in early phase studies of men with metastatic castration-resistant prostate cancer (mCRPC) that can reliably predict success in phase 3 trials. Among men with measurable disease, objective response may provide information as to whether a treatment is likely to be successful.

Methods

We conducted a systematic review of systemic agents that have proceeded to phase 3 trials in men with mCRPC and examined the relationship between improvements in measurable disease response in phase 2 trials and successful phase 3 trials leading to regulatory approval. Only trials that included men with radiographically measurable disease were included.

Results

We examined 31 eligible mCRPC phase 3 trials between 1992 and 2017 and 29 of the preceding phase 2 trials for RECIST responses. Measurable tumor responses in phase 2 trials were higher for successful therapies in phase 3 trials in chemotherapy-naive men with mCRPC, but were less correlated with success in trials investigating docetaxel combination regimens or the post chemotherapy mCRPC setting. Many failed agents did not produce higher than expected response rates over control arms; however, several agents such as anti-angiogenic therapies or orteronel produced higher than expected responses without survival benefit.

Conclusions

Objective responses in men with mCRPC may be associated with prolonged survival, but this association is mechanism dependent and inconsistent across trials or disease states. These data support considering RECIST response as a supportive but not sole endpoint in phase 2 trials to support launching phase 3 trials.

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Acknowledgements

This work was supported by the Duke Cancer Institute's NCI Core Grant P30 CA014236 and the Solid Tumor Therapeutics Program.

Funding

Outside of this work, AJA reports research support to Duke from Janssen, Medivation/Astellas/Pfizer, Bayer, Dendreon/SanPower, Sanofi Aventis, Genentech/Roche, Novartis; consulting with Janssen, Medivation/Astellas/Pfizer, Dendreon/San Power, Bayer; speaking for Dendreon/San Power, Bayer, Sanofi Aventis. GS reports consulting for Bayer, Sanofi, Pfizer, Novartis, Eisai, Janssen, Amgen, Astrazeneca, Merck, Genentech, Argos, Agensys, EMD Serono; Research support to institution from Bayer, Amgen-Onyx, Celgene, Boehringer-Ingelheim, Merck, Pfizer; Author for Uptodate; Speaker for Clinical Care Options, PER (Physicians Education Resource), RTP (Research To Practice), Onclive.

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  1. Department of Medicine, School of Medicine, Duke University, Durham, NC, USA

    Landon C. Brown & Andrew J. Armstrong

  2. Dana Farber Cancer Institute, Boston, MA, USA

    Guru Sonpavde

  3. Duke Cancer Institute and the Duke Prostate and Urologic Cancer Center, Duke University, Durham, NC, USA

    Andrew J. Armstrong

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Correspondence to Andrew J. Armstrong.

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Key Message In this systematic review of phase 2 and 3 trials in men with metastatic castration-resistant prostate cancer, objective radiographic responses may be associated with prolonged survival, but this association is mechanism dependent and inconsistent across trials or disease states.

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Brown, L.C., Sonpavde, G. & Armstrong, A.J. Can RECIST response predict success in phase 3 trials in men with metastatic castration-resistant prostate cancer?. Prostate Cancer Prostatic Dis 21, 419–430 (2018). https://doi.org/10.1038/s41391-018-0049-6

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  • DOI: https://doi.org/10.1038/s41391-018-0049-6

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