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.