Study Designs
The study designs in this section are summarized based on amended WRs rather than original WRs if the final study reports submitted were for trials conducted according to amended WRs. The solid tumor trials in 24 out of 27 WRs included both phase 1 and 2 studies. The trials in 2 WRs only included phase 2 studies because the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) in pediatrics had already established from trials conducted previously. Trials in 3 WRs were designed to have a tentative phase 2 study based on results from the phase 1 study. For those WRs that include trials for hematologic malignancies 12 out of 18 WRs included both phase 1 and 2 studies, trials in 2 WRs included only a phase 1 study, and trials in 4 WRs included a phase 2 study only.
For solid tumors, trials in 14 WRs have a control arm, with 3 used placebo with either best supportive care (BSC) or standard of care (SOC) and the rest 11 included active control arms. The other trials in 13 WRs are designed as single-arm. As a comparison, for hematologic malignancies, majority (15 out of 18) was single-arm design and only 3 have an active control.
As for treatment arm, trials in 23 WRs for solid tumor and 13 WRs for hematologic malignancies used monotherapy as the treatment arm; trials in 4 WRs for solid tumor and 5 WRs for hematologic malignancies used combination therapies.