Table 5 Final trial design parameters

Clinical parameters

DLT definition: cessation of IMP due to AE/AR

Dose set: 200, 400, 600, 800, 1000 mg daily

Starting dose: 400 mg

Fixed or variable sample size: fixed, n=21

Target level of DLT: 35%

Cohort size: 3

Model specification parameters

Model for dose-toxicity curve: Bayesian logistic regression

Initial MTD prior: 600 mg

One-stage or two-stage design: One

Estimation approach: Bayesian

Skeleton: (0.14, 0.23, 0.35, 0.47, 0.57)

Model: one parameter Bayesian model with prior distribution for slope N (μ = 0, σ = 0.265)

Practical considerations

Skipping doses: No

Early stopping rules: Yes

i) High probability (> 0.7) that posterior probability of DLT at lowest dose is greater than target DLT of 35%, indicating that the lowest dose is too toxic

ii) 4 consecutive cohorts allocated MTD, providing sufficient evidence MTD is reached