PNOC035: A platform study of novel therapies for children, adolescents and young adults with recurrent/progressive atypical teratoid rhabdoid tumor (ATRT)

What is the goal of the study?

Atypical teratoid rhabdoid tumor (ATRT) is a highly aggressive central nervous system (CNS) tumor that afflicts mostly young children (median age at diagnosis, two years). Less than 50% of patients will become long-term survivors despite intensive multi-modal therapies such as multi-agent chemotherapy, radiotherapy, high-dose chemotherapy with autologous stem cell rescue and intrathecal chemotherapy. Despite these advances, many patients still suffer recurrent disease with abysmal prognoses. Equally concerning is the long-term morbidity associated with the intensive treatments administered to the youngest ATRT patient population. Therefore, novel approaches for the treatment of ATRT are urgently needed to both improve patient survival and the quality-of-life of survivors. The Pediatric Neuro-Oncology Consortium (PNOC) ATRT working group has designed this study to provide a platform for the translation of novel therapies and combination strategies for recurrent ATRT with common primary objectives of safety and efficacy across the different arms. This platform study will offer phase 2 trials based on evolving preclinical data and clinical experience. When appropriate, there will be a safety lead-in/phase 1 component to investigate the incorporation of novel combinations and/or where the pediatric recommended phase 2 dose (RP2D) has not been determined. This platform design will allow for estimations of efficacy and toxicity for different treatment arms, allowing resource-sharing (regulatory infrastructure), thus shortening the timeline between different therapeutic options. STUDY ARMS: Arm A: Combination of Gemcitabine and Paxalisib

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team:

• PI (Study Doctor): Daniel Runco
• Study Team Member: HemOnc CRAs, [email protected]