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Research & Clinical Trials

Connecticut Children’s is leading the way in a broad collection of cutting-edge research. There are over 90 open protocols for treating and better understanding children’s cancer. In addition, our advanced cancer program provides access to cutting-edge, novel therapies for patients with resistant or recurring cancers. For patients looking for hope in a new treatment, we provide options close to home.

Advancing personalized medicine for children

We are pushing the boundaries for developing treatment plans based on whole genome analysis of a patient’s tumor. Our molecular-guided trials are working to expand the individual targeting of tumors through the use of surrogates (mice). We also anticipate that our partnership with The Jackson Laboratory in the area of genomic research will be transformational in the care of children with blood disorders and cancer.

Clinical trials

Connecticut Children’s Medical Center is one of about 220 members of the Children’s Oncology Group, an international consortium that seeks to cure or prevent childhood cancers through scientific discovery, and seeks to advance treatment for cancer through laboratory research and clinical trials. The Children’s Oncology Group has nearly 100 active clinical trials open at any given time, including front-line treatment for many types of childhood cancers, studies aimed at determining the underlying biology of these diseases, and trials involving new and emerging therapies, supportive care, and survivorship.

Clinical trials are studies conducted with voluntary patients to evaluate emerging cancer therapies, as well as innovative approaches to using combinations of existing therapies. There are three phases of clinical trials that are conducted before a new treatment is approved for general use:

  • Phase I – researchers test an experimental drug or treatment in a small group of people (20-80) to evaluate its safety, determine a safe dosage range and identify side effects.
  • Phase II – the experimental drug or treatment is given to a larger group of people (100-300) to test its effectiveness and further evaluate its safety.
  • Phase III – the drug or treatment is given to a larger group of people (1,000-3,000) to confirm its efficacy, monitor side effects, compare it to other commonly used treatments, and gather information that will and ensure it can be used safely.

Connecticut Children’s oncologists and hematologists believe that providing the best care for our pediatric patients means to offer them an opportunity to participate in clinical trials, from Phase I to Phase III. Our relationship with the Children’s Oncology Group helps us offer pediatric patients the latest innovations in clinical therapy, while also helping advance clinical cancer research that benefits children everywhere.

NIH-funded Research: Neuroblastoma

In 2012, Connecticut Children’s Medical Center was awarded a $2 million dollar grant from the National Institutes of Health-National Cancer Institute for a five-year study, “Sphingosine-1- Phosphate Pathway Based Therapy for Neuroblastoma.” The study investigates a novel approach to treating neuroblastoma, a deadly childhood tumor, by targeting a lipid signaling pathway essential to tumor growth.

Neuroblastoma is a cancerous tumor that develops from nerve tissue, typically occurring in infants and children. Extremely difficult to treat, in most patients Neuroblastoma has already spread when first diagnosed. It is most commonly identified in children before age five, and occurs in approximately one out of 100,000 children.

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