Design and rationale of FOCUS (PX-171-011): A randomized, open-label, phase 3 study of carfilzomib versus best supportive care regimen in patients with relapsed and refractory multiple myeloma (R/R MM)

Table 1 Study schema

Eligibility criteria	Randomization	Treatment arms	Follow-up
- Measurable multiple myeloma	Randomized 1:1	Carfilzomib	- Confirmed disease progression
- ECOG performance status 0-2	Stratified based on:	Cycle 1:	- Long-term follow-up assessments every 8 weeks until death or study closure
- ≥3 prior therapeutic regimens	- 3 vs 4 vs ≥ 5 therapies	20 mg/m² IV D 1,2
- Responsive to ≥1 line of therapy	- Europe vs non-Europe	27 mg/m² IV D 8, 9, 15, 16
- Relapsed while on or after 1 therapy		Cycle 2-9:
- Refractory to most recent regimen		27 mg/m² IV D 1, 2, 8, 9, 15, 16
		Cycle ≥10:
		27 mg/m² IV D 1, 2, 15, 16
		Best Supportive Care Regimen
		Prednisolone 30 mg QOD
		OR
		Dexamethasone 6 mg PO QOD
		plus optional
		Cyclophosphamide 50 mg PO QD
		(max 1400 mg/cycle)

D, day; ECOG, Eastern cooperative oncology group; IV, intravenous; PO, oral; QD, every day; QOD every other day.