Table I.

Major studies investigating ipilimumab [Yervoy®] (anti-CTLA-4 immunotherapy) to treat melanoma

Enrollment period	Trial phase/Identifier(s)	Patients	Randomization / Dosing regimen(s)	Primary endpoint(s) / Results	Median follow-up duration	Common severe (grade 3-5) irAEs:
2004-2008	Phase 3, MDX-010, NCT00094653	Previously treated, unresectable stage III or IV melanoma patients, n=676	Ipilimumab 3 mg/kg + gp100 every 3 weeks, for 4 treatments, n=403 Ipilimumab 3 mg/kg alone every 3 weeks for 4 treatments, n=137 gp100 alone every 3 weeks for 4 treatments, n=136	OS: Ipilimumab alone, 10.1 mo. Ipilimumab + gp100, 10 mo. gp100 alone, 6.4 mo.	Ipilimumab alone: 27.8 mo. Ipilimumab + gp100: 21 mo. gp100 alone: 17.2 mo.	Ipilimumab (+/− gp100): 10-15% gp100 alone: 3%
2008-2011	Phase 3, EORTC 18071, NCT00636168	Previously untreated resected stage III cutaneous melanoma patients, n=951	Ipilimumab, 10 mg/kg every 3 weeks for 4 doses; then every 3 months for up to 3 years, n=475 Placebo every 3 weeks for 4 doses; then every 3 months for up to 3 years, n=476	RFS: Ipilimumab: 26.1 mo. Placebo: 17.1 mo. 3-year RFS: Ipilimumab: 46.5% Placebo: 34.8%	2.74 years	Ipilimumab vs. placebo: GI: 16% vs. <1% Hepatic: 11% vs. <1% Endocrine: 8% vs. 0%

Abbreviations: glycoprotein 100 peptide vaccine (gp100); Overall survival (OS); Recurrence free survival (RFS)