Many cancers, such as osteosarcoma, neuroblastoma, and MRT/ATRT can be difficult to treat if they return after standard therapy. This clinical trial is testing a drug called panobinostat to see if it can help keep these tumours under control. Panobinostat is a type of “epigenetic” therapy, meaning it works by flipping “switches” inside cancer cells to slow down their growth. In this study, children and young adults receive a low daily dose of panobinostat as a pill for up to one year. The researchers are looking for the “sweet spot”: a dose that is strong enough to fight the cancer but gentle enough to minimise side effects. The team aims to find a new way to provide long term stability for patients with returning tumours.
This is an open label, Phase II, multicentre study evaluating the safety and anti tumour activity of continuous, low dose panobinostat in paediatric patients with refractory or recurrent solid tumours. Participants are stratified into three distinct histological cohorts: osteosarcoma, neuroblastoma, and malignant rhabdoid tumours/atypical teratoid rhabdoid tumours (MRT/ATRT). Panobinostat, a potent pan histone deacetylase (HDAC) inhibitor, is administered as a daily oral dose for up to 12 months. The trial utilises a dose titration design, starting at 10mg/m2, with adjustments based on real time biological effects and observed toxicities. The primary objective is to determine the Clinical Benefit Rate (CBR).