Table 1.

Treatment regimens

Stages and dosing schedule	Regimen	Formulation used	Cohort	Doses	Number of patients who received Dex*	Number of patients who received MRZ†
Stages 1-3: schedule A, once weekly	Days 1, 8, and 11, 4-wk cycles, 1 or 10 min	IV liquid	−3	0.025 mg/m2	0	3
			−2	0.05 mg/m2	0	3
			−1	0.075 mg/m2	0	7
			1	0.15 mg/m2	0	2
			2	0.3 mg/m2	0	1
			3	0.6 mg/m2	0	3
			4/RP2D§	0.7 mg/m2	0	8
		IV lyophile‡	Expanded RP2D*,§	0.7 mg/m2	0	5
Stages 4 and 5: schedule B, twice weekly	Days 1, 4, 8, and 11, 3-wk cycles, 1 or 2 h	IV lyophile	1	0.15 mg/m2	1	3
			2	0.4 mg/m2	3	4
			3	0.5 mg/m2	1	2
			4	0.6 mg/m2	3	3
			RP2D§	0.5 mg/m2	15	24

^*Dex, if administered, was instructed to be administered on the day of and the day after MRZ dosing.

^†Data from study NPI-0052-102 provided safety information to allow some dose levels (0.15 mg/m² and 0.3 mg/m²) to be bypassed with fewer than 3 patients enrolled.

^‡On schedule A, all patients who were dosed prior to the RP2D cohort received the liquid formulation; the 5 schedule A patients in the RP2D cohort received the lyophile formulation by crossover (ie, alternate assignment of patients to receive their first dose using either the lyophile or liquid formulation).

^§The schedule A RP2D was established as 0.7 mg/m² infused over 10 minutes, and the schedule B RP2D was 0.5 mg/m² infused over 2 hours.