Research And Grants

CONNECT Consortium - $489,801.26

CONNECT Consortium – Nationwide Children’s Hospital
Grant Amount



September 2020

Research Type


Cancer Type


CONNECT (COllaborative Network for NEurooncology Clinical Trials) Consortium

CONNECT’s goal is to establish an international clinical trials collaboration for the conduct of scientifically rational, pilot studies to assess feasibility and early efficacy of incorporating promising novel agents to established frontline therapeutic regimens in children with newly-diagnosed, high-risk brain tumors. This collaboration builds on the existing infrastructure and partnerships established through the International DIPG Registry and SIOPE Registries.

Pediatric NCI-funded phase I consortia (e.g. COG DVL and PBTC) as well as non-NCI funded consortia (e.gPNOC, POETIC and ITCC) have helped to ensure that novel agents undergo phase I testing to define toxicities and preliminary pharmacokinetic and pharmacodynamics endpoints in children with recurrent tumors. Similarly the Children’s Oncology Group and the SIOPE disease networks, as well as other national consortia, have played critical role in conducting large phase II/III trials to establish current therapeutic standards in frontline regimen for children with CNS malignancies. However, phase II/III trials require the commitment of extensive time and resources to demonstrate improved outcomes. As our expanding knowledge of biological targets points to numerous potential targeted agents, judicious decisions must be made before larger efficacy studies are conducted incorporating novel agents in the frontline setting.

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 and therapeutic modalities into the most effective current regimens for children with newly-diagnosed high-risk brain tumors. To accomplish this goal CONNECT will leverage the considerable strength in basic science and translational expertise among its global collaborative partners. These sites all have a well-deserved reputation for robust clinical research programs, with strong leadership and expertise in early phase clinical trials, pharmacology, translational research, and imaging. A preclinical initiative among member institutions will assess promising agents in a broad array of preclinical disease models to fast-track development of promising experimental agents in high-risk pediatric brain tumors. The neuropathology, neuroimaging, and genomics cores will develop and support rational inquiries and correlative studies that will lead to scientific discovery and inform future clinical trials. Sixteen institutions have agreed to work together to conduct testing of novel agents in high-risk, poor-prognosis pediatric CNS tumors such as diffuse intrinsic pontine glioma (DIPG), high-grade gliomas (HGG), atypical teratoid rhabdoid tumor(ATRT), and high-risk medulloblastoma (MB). All of these centers have been active members of the International or SIOPE DIPG Registries and have a proven track record of working effectively as collaborators. All sites have expertise in conducting early phase and complex clinical trials and are members and leaders of other key consortia such as COG, PBTC, DVL, PNOC, ITCC, and SIOPE.

Studies will be conducted in small cohorts of patients to establish safety and tolerability as well as to detect early efficacy of novel, intensified regimens in order to accelerate development of promising regimens. CONNECT will work collaboratively to translate feasible, tolerable and promising novel regimens in children with newly-diagnosed, high-risk, poor-prognosis CNS malignancies to larger consortia with appropriate infrastructure to conduct definitive phase II and III trials (e.g. COG, SIOPE, others).

Related funding:

  • CONNECT2007 - Phase I/II Study of Lutathera in Pediatric and Young Adult Patients with Recurrent and/or Progressive High-Grade Central Nervous System (CNS) Tumors and Meningiomas which Express Somatostatin Type 2A Receptors and Demonstrate Uptake on DOTATATE
  • CONNECT2109A - A molecularly-guided phase II study of ribociclib and everolimus in children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG) and high-grade glioma (HGG)
  • Immuno-Modulation to Treat Poor-Prognosis Pediatric Brain Tumors