by conducting nimble, scientifically-rational, international trials to demonstrate the safety and feasibility of incorporating novel agents and therapeutic modalities into the most effective current regimens for children with newly-diagnosed high-risk brain tumors.
The COllaborative Network for NEuro-oncology Clinical Trials (CONNECT) conducts clinical trials in high-risk pediatric brain tumors (such as DIPG) to investigate combinations of novel drugs with traditional therapies. We seek to improve outcomes in the worst childhood brain tumors by combining innovative discoveries with “tried and true” therapy regimens through:
CONNECT member institutions lead the way in cutting edge research in the biology of high-risk pediatric brain tumors. By combining the considerable strengths in basic science and translational expertise among CONNECT’s international members, we can quickly translate basic science discoveries into novel therapies and new cures.
CONNECT is a global partnership of world-class childhood cancer centers and pediatric neuro-oncology experts. The international collaboration expands global access to novel, promising therapies for children and young adults with brain tumors, and improves scientific discovery through collaboration
In this research study the investigators want to learn more about the safety of the study drug, PTC596 has when taken during radiation. The investigators also want to learn about the effects, if any, these drugs have on children and young adults with brain tumors.
The investigators are asking people to be in this research study who have been diagnosed with a high grade glioma (HGG) including diffuse intrinsic pontine glioma (DIPG) to be in the research, because they have scheduled to have radiation to treat their cancer.
The study is divided into two parts. The goal of the first part is to find the dose of PTC596 that can be given with radiation without causing serious side effects. The purpose of this surgical study is to test the amount of a study drug that may be found in the tumor and blood when given prior to and during a planned surgery for removal of the recurrent tumor.
In this research study, we want to learn about the safety of the study drugs, ribociclib and everolimus, when given together at different doses after radiation therapy. We also want to learn about the effects, if any, these drugs have on children and young adults with brain tumors.
We are asking people to be in this research study who have been diagnosed with a high grade glioma, their tumor has been screened for the Rb1 protein, and they have recently finished radiation therapy. If a patient has DIPG or a Bi-thalamic high grade glioma, they do not need to have the tumor tissue screened for the Rb1 protein, but do need to have finished radiation therapy.
Tumor cells grow and divide quickly. In normal cells, there are proteins that control how fast cells grow but in cancer cells these proteins no longer work correctly making tumor cells grow quickly. Both study drugs work in different ways to slow down the growth of tumor cells. The researchers think that if the study drugs are given together soon after radiation therapy, it may help improve the effect of the radiation in stopping or slowing down tumor growth.
The study drugs, ribociclib and everolimus, have been approved by the United States Food and Drug Administration (FDA). Ribociclib is approved to treat adults with breast cancer and everolimus is approved for use in adults and children who have other types of cancers. The combination of ribociclib and everolimus has not been tested in children or in people with brain tumors and is considered investigational.