Table 2.

Recommended lupus trial designs

Phase	Robustness (discriminatory capacity)	Size (patient no)	Description	Duration (months)
Ib/2a	Very high	60–100	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
	High	100–200	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 design	60–200	Adaptive Trial: Example of PERFECT design. 80 Exploratory pharmacodynamics optimise dosing or determine continuation to efficacy endpoint	12
2b/3	High	150–300	Based on Anifrolumab Ph 2 design. 12 Require steroid taper as tolerated in patients with moderate to moderately severe disease	6–12
	High	150–300	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 design	150–300	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