A PHASE II STUDY OF PANOBINOSTAT IN PAEDIATRIC PATIENTS WITH SOLID TUMORS INCLUDING MALIGNANT RHABDOID TUMOR/ATYPICAL TERATOID RHABDOID TUMOURS

Abstract

Low-dose panobinostat treatment has been shown to terminally differentiate malignant rhabdoid tumor (MRT)/atypical teratoid rhabdoid tumors (ATRT) in pre-clinical models. This will be an open label, phase II study, of panobinostat in patients with newly diagnosed MRT/ATRT, aimed to define the anti-tumour activity of panobinostat. To define the anti-tumour activity of low dose continuous panobinostat. To define and describe the associated toxicities of panobinostat. To define the anti-tumor activity of low dose, continuous panobinostat using functional imaging techniques. To assess the biologic activity of low dose panobinostat by measuring histone acetylation status in peripheral mononuclear..