Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT01660984 |
| Other study ID # |
120178 |
| Secondary ID |
12-C-0178 |
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
July 30, 2012 |
Study information
| Verified date |
April 24, 2024 |
| Source |
National Institutes of Health Clinical Center (CC) |
| Contact |
Nicole Lucas |
| Phone |
(240) 760-6252 |
| Email |
andrea.lucas[@]nih.gov |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
Background:
- Medullary thyroid cancer (MTC) is a rare cancer of the thyroid gland. In children and
adults, it is often part of a condition called Multiple Endocrine Neoplasia 2 (MEN2). MEN2 is
usually caused by a genetic mutation, and it can cause a number of problems in addition to
MTC. These problems include adrenal gland tumors, hormone changes, and problems with the
bones and other organs. Not much is known about how MTC develops over time, especially in
people with MEN2. Researchers want to study MTC in children and adults and see how it affects
their growth and development.
Objectives:
- To study how medullary thyroid cancer affects children and adults over time.
Eligibility:
- Children and adults who have medullary thyroid cancer.
Design:
- Participants will be screened with a brief physical exam and medical history. Blood and
tissue samples will be collected to see whether participants have the MEN2 genetic
mutation.
- Treatment will not be provided as part of this study. However, participants will be
receiving standard care for MTC. They may be eligible for other clinical trials at the
National Institutes of Health.
- Participants will have regular study visits every 6 to 12 months to evaluate their MTC
and any treatment. Blood tests, imaging studies, and other tests may be performed as
needed to monitor the disease.
- Participants and their parents/guardians will also complete questionnaires about their
health and emotions during the study.
Description:
Background:
- Medullary Thyroid Carcinoma (MTC) is a calcitonin producing tumor arising from the
parafollicular C cells of thyroid. In children and 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 MEN2 with or without MTC, particularly in children and 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.
Eligibility:
- Patients, must have histologically or cytologically confirmed MTC, confirmed by the
Laboratory of Pathology, NCI or who have MEN2 (regardless of MTC status).
- Parent or primary caregiver of patient participant (<= 21)
- Participants may be undergoing standard care or receiving treatment on a clinical trial
while participating in this study.
Design:
This study will allow for longitudinal evaluations of MTC and MEN2 and non-tumor related
manifestations of MEN 2A and 2B in children and adults. Evaluations will consist of the
following (summarized):
1. Clinical and radiological evaluations
2. Detailed pathologic and molecular analysis of tumor specimens will be performed,
including immunohistochemistry (IHC), comparative genomic hybridization (CGH), and
genome sequencing. CGH and genome sequencing will be performd with co-enrollment on
protocol 10-C-0086 Comprehensive Omics Analysis of Pediatric Solid Tumors and
Establishment of a Repository for Related Biological Studies.
