Table 3.

Phase II–III trials for lymphomas

ObjectivesRecommendations
Phase II
  • –Evaluate activity of single agents before moving to phase III trials

  • –DCR encompassing CR+PR+minor tumor responses may be a clinically meaningful endpoint

  • –Evaluation of patients' subsets and biomarkers to be implemented in future phase II trials

  • –Evaluation of new drug combinations

  • –Priority should be given to testing combinations that are based on strong preclinical rationale

  • –A high efficacy bar in terms of responses or time-to-event endpoints should be used before moving to phase III trials

  • –New drugs with modest single agent activity may be tested in combination with standard regimens

  • –Phase II trials may be skipped for drugs with established single-agent activity when studying a combination with established regimes following safety evaluation in phase Ib studies

  • –Multi-arm trials testing different agents or one agent against different lymphoma types may help to identify a subset of patients that benefit from a new drug or a new combination

Phase III
Efficacy evaluation of a new drug or new drugs combination against established regimen
  • –Exploratory endpoints such as evaluation of minimal residual disease should be added to traditional endpoints

  • –In DLBCL, PFS at 24 months is proposed as a surrogate for OS

  • –Adaptive designs may reduce the time for completing phase III trials