Results of an International Randomized Phase III Trial of the Mammalian Target of Rapamycin Inhibitor Ridaforolimus Versus Placebo to Control Metastatic Sarcomas in Patients After Benefit From Prior Chemotherapy

摘要: Purpose Aberrant mammalian target of rapamycin (mTOR) signaling is common in sarcomas and other malignancies. Drug resistance toxicities often limit benefits systemic chemotherapy used to treat metastatic sarcomas. This large randomized placebo-controlled phase III trial evaluated the mTOR inhibitor ridaforolimus assess maintenance disease control advanced Patients Methods with soft tissue or bone who achieved objective response stable prior were randomly assigned receive 40 mg placebo once per day for 5 days every week. Primary end point was progression-free survival (PFS); secondary points included overall (OS), best lesion response, safety, tolerability. Results A total 711 patients enrolled, 702 received blinded study drug. Ridaforolimus treatment led a modest, although significant, improvement PFS independent review compared (hazard rat...