Study Designs
The study designs in this section are summarized based on amended WRs rather than original WRs if the studies completed and submitted were conducted according to amended WRs. The studies in 24 out of 29 solid tumor WRs have combined phase 1 and 2 studies. The studies in 2 WRs only included phase 2 studies because the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) in pediatrics was already established from trials conducted previously. Studies in 3 WRs were designed to have a tentative phase 2 study based on results from the phase 1 study. For hematologic malignancy WRs, studies in 12 out of 18 WRs conducted combined phase 1 and 2 studies, studies in 2 WRs conducted only a phase 1 study, and studies in 4 WRs conducted a phase 2 study only.
For solid tumors, studies 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 15 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, studies in 25 WRs for solid tumor and 13 WRs for hematologic malignancies used monotherapy as the treatment arm; studies in 4 WRs in solid tumor and 5 WRs in hematologic malignancies used combination therapies.