Table 1.

Carfilzomib phase II clinical trial characteristics

Study	MM status	Prior therapy	Carfilzomib dosinga	N (total)	N (used in model development)	N (observations)	Reference
PX‐171‐003‐A0	Relapsed and refractory	≥2 regimens; responded to first‐line and refractory to most recent	20 mg/m2	43	39	152	21
PX‐171‐003‐A1b	Relapsed and refractory	≥2 regimens; responded to ≥1 and refractory to most recent	20/27c mg/m2	259	235	1393	22
PX‐171‐004	Relapsed and/or refractory	Responded to first‐line; relapsed or refractory to ≥1 but ≤3 regimens	20 or 20/27c mg/m2	162	146	1088	23,24
PX‐171‐005	Relapsed and/or refractory with various levels of renal insufficiency	≥2 regimens; achieved ≥MR to ≥1	15, 20, 27d mg/m2	49	36	102	25
Total				513	456	2735

MM, multiple myeloma; MR, minimal response; N, number of subjects or observations.

^aDays 1, 2, 8, 9, 15, and 16 of a 28‐day cycle.

^bStudy PX‐171‐003‐A1 is the pivotal trial that provided response data that supported an accelerated approval of the US Food and Drug Administration for carfilzomib in the United States.

^c20 mg/m² in cycle 1, and then 27 mg/m² thereafter.

^dIncreased each cycle as tolerated.