Table 4.

Key trials in the development of the neurokinin (NK)₁-receptor antagonist aprepitant

References	Design	Investigational arm	Comparator	Chemotherapy	Primary parameter	Result
Navari et al. [97]	R, DB, P n = 158	All: GRA 10 μg/kg IV + DEX 20 mg PO on day 1 a) APR 400 mg PO on day 1 +  APR 300 mg PO on days 2–5 b) APR 400 mg PO on day 1 c) PLA	GRA 10 μg/kg IV + DEX 20 mg PO on day 1 + PLA (arm c)	CIS	No EE, 25–120 h	APR > PLA
Chawla et al. [101]	R, DB, P n = 381	All: OND 32 mg IV + DEX 20 mg PO on day 1 + DEX 8 mg PO on days 2–5 a) APR 40 mg PO on day 1 + APR 25 mg PO on days 2–5 b) APR 125 mg PO on day 1 + APR 80 mg PO on days 2–5	OND 32 mg IV + DEX 20 mg PO on day 1 + DEX 8 mg PO on days 2–5 + PLA on days 1–5	CIS	Complete response (no EE, no rescue medication) 0–120 h	Both APR doses > PLA PLA 125/80 chosen for phase III studies (3-day regimen)
Hesketh et al. [102]	R, DB, P n = 521	APR 125 mg PO + OND 32 mg IV + DEX 12 mg PO on day 1 + APR 80 mg PO + DEX 8 mg PO on days 2–3 + DEX 8 mg PO on day 4	OND 32 mg IV + DEX 20 mg PO on day 1 + DEX 8 mg × 2 PO on days 2–4	CIS	Complete response (no EE, no rescue medication) 0–120 h	APR > PLA
Poli-Bigelli et al. [103]	R, DB, P n = 567	APR 125 mg PO + OND 32 mg IV + DEX 12 mg PO on day 1 + APR 80 mg PO + DEX 8 mg PO on days 2–3 + DEX 8 mg PO on day 4	OND 32 mg IV + DEX 20 mg PO on day 1 + DEX 8 mg × 2 PO on days 2–4	CIS	Complete response (no EE, no rescue medication) 0–120 h	APR > PLA
Warr et al. [105]	R, DB, P n = 866	APR 125 mg PO + OND 8 mg × 2 PO + DEX 12 mg PO on day 1 + APR 80 mg PO on days 2–3	OND 8 mg ×2 PO + DEX 20 mg PO on day 1 + OND 8 mg × 2 PO on days 2–3	AC-based	Complete response (no EE, and no rescue medication) 0–120 h	APR > PLA

A anthracycline, APR aprepitant, C cyclophosphamide, CIS cisplatin, DB double-blind, DEX dexamethasone, EE emetic episodes, GRA granisetron, IV intravenously, OND ondansetron, P parallel, PLA placebo, PO orally, R randomized