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Treating Hereditary Thyroid Cancer in Children

Name of the Trial

Phase I/II Study of Vandetanib in Young Patients With Hereditary Medullary Thyroid Carcinoma (NCI-07-C-0189). See the protocol summary.

Principal Investigators

Dr. Frank Balis, NCI Center for Cancer Research, and Dr. Samuel Wells, Washington University, St. Louis.

Dr. Frank Balis

Dr. Frank Balis Principal Investigator

Why This Trial Is Important

Medullary thyroid carcinoma (MTC) is a rare type of thyroid cancer, representing about 2-3 percent of thyroid cancer cases. Although MTC usually occurs in people who have no family history of the disease, about 25-40 percent of cases are hereditary. Hereditary MTC is associated with group of genetic disorders that are caused by inherited mutations in a gene called RET. People with these disorders usually develop MTC as children or young adults.

Surgery is the only curative therapy for patients with hereditary MTC. Patients whose tumors cannot be surgically removed (unresectable) or that recur following surgery need new treatment options.

In this trial, doctors are testing a new drug called vandetanib (Zactima) in young patients with advanced hereditary MTC. Vandetanib blocks the activity of the protein produced by the RET gene. Researchers believe that inhibiting the activity of the RET protein may cause tumors to shrink and reduce the levels of tumor biomarkers in patients with MTC. Besides looking for these treatment effects, doctors will assess the safety and the pharmacokinetics of vandetanib in these patients.

"MTC is the most common cause of death in patients with these genetic disorders, and the disease is relatively unresponsive to radiation therapy and standard or novel chemotherapeutic regimens," said Dr. Balis. "Vandetanib, unlike standard chemotherapy or radiation, specifically targets the genetic defect responsible for the development of these tumors."

Who Can Join This Trial

Researchers seek to enroll 21 patients aged 5 to 18 with hereditary MTC that cannot be removed surgically, has recurred, or has metastasized (spread). See the list of eligibility criteria.

Study Site and Contact Information

This study is taking place at the NIH Clinical Center in Bethesda, Md. For more information, call the NCI Clinical Trials Referral Office at 1-888-NCI-1937. The toll-free call is confidential.

  • Posted: December 18, 2007