CONNECT is an international collaborative network of pediatric cancer centers with the objective to improve outcomes for children with newly-diagnosed, high-risk brain tumors (including diffuse intrinsic pontine glioma, high-grade gliomas, atypical teratoid rhabdoid tumors, and high-risk medulloblastoma). We conduct small, scientifically-rational, pilot studies to assess feasibility and early efficacy of incorporating promising new therapies into established frontline therapeutic regimens. CONNECT works alongside phase I consortia and large clinical trial groups to target newly-diagnosed tumors.
Large pediatric cancer consortia have helped to ensure that novel agents undergo rigorous phase I testing to define toxicities in children with recurrent tumors. However, these studies evaluate new agents alone and in isolation, without combining them with traditional therapies. Similarly, large national consortia (such as COG and SIOPE), have played a critical role in conducting large phase II/III trials that test the current therapeutic standards. However, phase II/III trials require enormous amounts of time and extensive resources. Understanding whether the combination of a new therapy with an established frontline therapy regimen is tolerable in children would help guide these large studies as they are designed.
CONNECT’s goal is to fill this critical gap by conducting nimble, scientifically-rational, international trials to demonstrate the safety and feasibility of incorporating novel agents into the most effective current regimens for children with newly-diagnosed high-risk brain tumors.
Studies will be conducted in small groups of pediatric patients to establish that combination therapy is safe and tolerable in children, as well as to detect early signs that combined therapy may be more effective than traditional therapy regimens. Once combinations are shown to be safe and effective, CONNECT works collaboratively with larger consortia to help move these discoveries into larger trials.