A Phase II Trial of Guadecitabine in Children and Adults with SDH-Deficient GIST, Pheochromocytoma, Paraganglioma, and HLRCC-associated Renal Cell Carcinoma.
John A LigonR Taylor SundbyMary Frances Wedekind MaloneFernanda I ArnaldezJaydira Del RiveroLori S WienerRamaprasad SrinivasanMelissa SpencerAmanda CarbonellHaiyan LeiJack F ShernSeth M SteinbergWilliam Douglas FiggCody J PeerSara M ZimmermanJosquin MoralyXia XuStephen FoxKing ChanMichael I BarbatoThorkell AndressonNaomi TaylorKarel PacakJ Keith KillianEva DombiW Marston LinehanMarkku M MiettinenRichard L PiekarzLee J HelmanPaul S MeltzerBrigitte C WidemannJohn W GlodPublished in: Clinical cancer research : an official journal of the American Association for Cancer Research (2022)
Guadecitabine was tolerated in patients with dSDH tumors with manageable toxicity. While 4/9 patients had prolonged stable disease there were no objective responses. Thus, guadecitabine did not meet the target of 30% response rate across dSDH tumors at this dose, though signs of biologic activity were noted.