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Understanding 

 treatment options

For children with neuroblastoma, sarcomas and brain tumors, clinical trials offer access to new and novel treatment options. By enrolling in a clinical trial, children whose cancer has returned or whose cancer does not respond to treatment may benefit from a new experimental drug or therapy.

Phase I trials often include a small number of children (10 to 30) and test novel therapies for safety, toxicity and dose. Phase I trials are exclusively for children whose cancer has stopped responding to treatment and children whose cancer has returned.

Phase II trials test the efficacy of a new treatment in specific cancers and enroll 30-60 children. Some phase II are randomized to test the difference between new therapies. Phase II trials are exclusively for children whose cancer has stopped responding to treatment and children whose cancer has returned.

Phase III trials compare the current best treatment with a new therapy for a specific cancer in children who are newly diagnosed. Phase III trials may combine chemotherapy, radiation or surgery with a new treatment. Phase III trials enroll a large number of patients and may include randomization to determine the specific treatment option that your child receives.

finding a clinical trial for your child

If your child’s cancer is not responding to treatment or if the cancer has returned after a period of remission, your doctor may recommend a clinical trial. We understand the urgency that you may feel to consider many additional options and research different clinical trials.

Visit www.clinicaltrials.gov to find the most comprehensive list from the National Institutes of Health.

Prepare a treatment summary. You may need key information about your child’s medical history to find a suitable trial. Your doctor can provide a treatment summary for you, but you may want your own copy to keep it up to date.

Your child’s treatment summary should include:
a. Diagnosis details, age, tumor type, etc.
b. All treatment and dates received
c. Copies of scans and results
d. Current health and disease status

Talk to your doctor. It’s a good idea to include your pediatric oncologist in any conversation about your child’s treatment. Ask your child's doctor about clinical trial options and about specific trials that you may have come upon in your research. If you are not satisfied with your oncologist’s treatment approach, you can always seek a second opinion.

Contact the Principal Investigator (PI) of the trial you are interested in.  After talking with your pediatric oncologist, if a specific clinical trial may be reasonable to consider, you can ask your doctor to contact the PI or you can contact the PI yourself. You may be asked to send your child’s treatment history in advance and follow-up with questions later about the trial.