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Thyroid Cancer

Longitudinal Assessment and Natural History Study of Children and Young Adults With Medullary Thyroid Carcinoma

NCI-12-C-0178 , NCT01660984

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Investigator(s):

Brigitte Widemann, M.D.
Principal Investigator
Phone: 301-496-7387
widemanb@mail.nih.gov

Referral Contact(s):

Pediatric Oncology
Phone: 301-496-4256
1-877-624-4878 (Toll free)

 

Background:

  • Medullary thyroid carcinoma (MTC) is a calcitonin producing tumor arising from the parafollicular C cells of the thyroid
  • In children and young adults, MTC is usually seen in association with multiple endocrine neoplasia (MEN) 2A and 2B, which are rare cancer syndromes resulting from germline mutations of rearranged during transfection (RET) proto-oncogene
  • MTC develops in virtually all patients with MEN 2 and is the leading cause of death in these patients; patients with MEN 2 may have other characteristic manifestations such as pheochromocytoma and hyperparathyroidism in MEN 2A and pheochromocytoma, ganglioneuromatosis, and skeletal deformities in MEN 2B
  • Complete surgical resection is the only current curative treatment for MTC, and the tumor is unresponsive to standard chemotherapy and conventional doses of radiation therapy; however, more than half the patients present with advanced or metastatic disease and cannot be cured surgically
  • Novel agents are currently under investigation for treatment of MTC, and vandetanib, an oral RET and receptor tyrosine kinase (RTK) inhibitor was recently approved by the FDA for adults with advanced or metastatic MTC; vandetanib also has activity in children with hereditary MTC
  • However, complete responses to RTKs have not been observed, and some patients develop resistance to the treatment with RET and RTK inhibitors or have primary refractory disease; the natural history of MTC, particularly in patients with MEN 2, the molecular pathways involved in tumorigenesis, and the development of resistance to targeted therapies are not well understood

Objectives:

  • The overall objective of this longitudinal study is to develop a better understanding of the biology and natural history of MTC, particularly in children and young adults with MEN 2A and 2B, as well as study non-tumor manifestations of MEN 2
  • This will hopefully allow for developing more effective treatment interventions for tumor and non-tumor related manifestations and more sensitive endpoints in clinical trials

Key Eligibility Criteria:

  • Patients ≤ 25 years of age, must have histologically or cytologically confirmed MTC confirmed by the Laboratory of Pathology, NCI
  • Participants may be undergoing standard care or receiving treatment on a clinical trial while participating in this study

Study Outline:

  • This study will allow for longitudinal evaluations of MTC and non-tumor related manifestations of MEN 2A and 2B in children and young adults; evaluations will consist of the following (summarized):
    • Clinical and radiological evaluations every 6–12 months
    • Detailed pathologic and molecular analysis of tumor specimens will be performed, including immunohistochemistry (IHC), comparative genomic hybridization (CGH), and genome sequencing

Additional Information:

  • This trial will be conducted at the NIH Clinical Center in Bethesda, MD. It is open to patients who meet the eligibility requirements, regardless of where they live in the United States.
  • There is no charge for medical care received at NIH Clinical Center.
  • PDQ (Physicians Data Query) - provides additional details about this study for health care providers.


Reviewed: 12/14/12
Updated: 12/27/12

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