Design and Endpoints of Clinical Trials, Current and Future
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With the advent of several new systemic agents for the treatment of hepatocellular carcinoma and the prospect of more to come it is expected that many more clinical trials will be undertaken to establish the best treatment paradigm(s). In order to help develop the most efficient and most relevant clinical trials this review concentrates on endpoints that have been used in the past. Survival is the gold standard. None of the surrogate endpoints correspond completely with survival. In addition, alternative clinical trial designs are presented that may be more efficient than the usual phase I, II, and III clinical trial strategy that has been used in the past.
KeywordsHepatocellular carcinoma Clinical trial design Clinical trial endpoints
Compliance with ethical standards
Conflict of interest
The author declare that they have no conflict of interest.
- 4.Zhu, AX, Kang YK, Yen CJ, Finn RS, Galle PR, Llovet JM. REACH-2: a randomized, double-blind, placebo-controlled phase 3 study of ramucirumab versus placebo as second-line treatment in patients with advanced hepatocellular carcinoma (HCC) and elevated baseline alpha-fetoprotein (AFP) following first-line sorafenib. J Clin Oncol. 2018;36:4003.Google Scholar
- 5.Abou-Alfa GK, Meyer T, Cheng AL, El-Khoueiry A, Rimassa L, Ryoo BY. Cabozantinib versus placebo in patients) with advanced hepatocellular carcinoma who have received prior sorafenib: results from the randomized phase 3 CELESTIAL trial. J Clin Oncol. 2018;36:4019.Google Scholar
- 19.Zhang BH, Yang BH, Tang ZY. Randomized controlled trial of screening for hepatocellular carcinoma. J Cancer Res Clin Oncol. 2004;130:417–422.Google Scholar
- 21.Mandrekar SJ, Dahlberg SE, Simon R. Improving clinical trial efficiency: thinking outside the box. Am Soc Clin Oncol Educ Book. 2015. https://doi.org/10.14694/EdBook_AM.2015.35.e141.