Table 2

Recommended lupus trial designs

PhaseRobustness
(discriminatory capacity)
Size
(patient no)
DescriptionDuration (months)
Ib/2aVery high60–100 Based on BOLD design. 77
Withdraw or quickly taper all background medications in an active, mild–moderate population without risk for organ-threatening disease
6–8
High100–200 Based on Anifrolumab Ph 2 design. 12 Require steroid taper as tolerated in patients with moderate to moderately severe disease.6–12
Increases efficiency of any design60–200 Adaptive Trial: Example of PERFECT design. 80 Exploratory pharmacodynamics optimise dosing or determine continuation to efficacy endpoint12
2b/3High150–300 Based on Anifrolumab Ph 2 design. 12 Require steroid taper as tolerated in patients with moderate to moderately severe disease6–12
High150–300 Based on Classic Design but restricted to patients with more severe disease. 76 No medication taper required. An optimal design would select background treatments based on rational pharmacodynamics.6–12
Increases efficiency of any design150–300 Adaptive Trial 79: Here Pharmacodynamic exploration is not part of trial , but dosing adjustments and/or primary endpoint may be adjusted based on clinical or biomarker-driven data (patients who fail screening can be retained for secondary endpoints or safety).12