Longitudinal Assessment and Natural History of Younger Patients with Medullary Thyroid Cancer and Multiple Endocrine Neoplasia 2 and Their Caregivers

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Basic Trial Information

PhaseTypeAgeTrial IDs
No phase specifiedBiomarker/Laboratory analysis, Natural history/Epidemiology25 and under12-C-0178
NCI-2015-01255, 321080, P12989, NCT01660984

Trial Description

Summary

This pilot research trial follows patients over a long period of time (longitudinal assessment) and studies the natural history of younger patients with medullary thyroid cancer (MTC) and multiple endocrine neoplasia 2 (MEN2) and their caregivers. MEN2 is a rare, genetic disorder that affects the endocrine glands and can cause tumors in the thyroid, parathyroid, or adrenal glands. Learning about the natural history of these conditions (knowledge about a disease and its course over time) and the biology of the tumors (how they develop, grow, spread, and respond to treatment), may help doctors better understand these conditions, which may lead to more effective treatments.

Further Study Information

PRIMARY OBJECTIVES:

I. To develop a better understanding of the biology and natural history of MTC, particularly in children and young adults with multiple endocrine neoplasia 2 (MEN 2).

II. Study longitudinally the growth rate of primary and metastatic tumor lesions in children and young adults with MTC, irrespective of whether they are receiving tumor directed treatment.

III. Study molecular pathways altered in MTC and their relationship to the patients’ clinical course and response to targeted therapies.

SECONDARY OBJECTIVES:

I. Describe the non-tumor related manifestations of MEN 2 including skeletal, endocrine, and gastrointestinal manifestations, and their clinical course over time.

II. Evaluate the role of fludeoxyglucose F 18 (FDG) positron emission tomography (PET) computed tomography (CT)/magnetic resonance imaging (MRI) in the detection and monitoring of MEN 2 associated tumors.

III. Investigate the psychosocial experiences and needs of persons living with MTC and their caregivers, and assess how these change over time.

OUTLINE:

Patients undergo history and physical examination, including evaluation of growth and sexual maturation, pheochromocytoma, skeletal abnormalities, gastro-intestinal and nutritional manifestations, mucosal neuromas, performance status; medical photography (optional); ret proto-oncogene (RET) mutation analysis; and undergo collection of tissue samples for molecular analysis. Patients also undergo FDG-PET/CT and FDG PET/MRI and bone scan if clinically indicated. Parents and/or caregivers and patients 12 years and older undergo evaluation of psychosocial experiences and needs.

After completion of study, patients are followed up for 5 years.

Eligibility Criteria

Inclusion Criteria:

Ability to travel to the National Institutes of Health (NIH) and to undergo evaluations to be performed on this protocol

For the purpose of this study subjects who have not previously received medical or surgical treatment, patients who have previously received medical or surgical treatment, and subjects who are currently receiving medical treatment and or radiation for a MEN 2 related manifestations will be eligible; prior and current treatment for MEN 2 related manifestations will be recorded at trial entry and throughout the study

Patients must have a primary care provider (for example a primary oncologist or endocrinologist) who can provide and coordinate the medical care for the patient

All patients or their legal guardians (if the patients is < 18 years old) must sign an Institutional Review Board (IRB)-approved document of informed consent to demonstrate their understanding of the investigational nature and the risks of this study before any protocol-related studies are performed; when appropriate, pediatric subjects will be included in all discussions

Patients must have histologically or cytologically confirmed MTC, confirmed by the Laboratory of Pathology, National Cancer Institute (NCI)

Patients must have evidence of active MTC as documented by either elevated plasma biomarkers (carcinoembryonic antigen [CEA] and/or calcitonin), or presence of evaluable or measurable disease

Exclusion Criteria:

In the opinion of the investigator the patient is not able to return for follow-up visits or obtain required follow-up studies

Individuals who are pregnant or breast feeding or who become pregnant while enrolled on this trial will not be excluded from participation, but will not undergo radiographic evaluations or other studies for research purposes

Trial Contact Information

Trial Lead Organizations / Sponsors / Collaborators

NCI - Center for Cancer Research

  • National Cancer Institute
Brigitte C. Widemann, Principal Investigator

Trial Sites

U.S.A.

Maryland
Bethesda

Mark O Hatfield-Warren Grant Magnuson Clinical Center

Brigitte C. Widemann
Ph: 301-496-7387
Email: widemanb@mail.nih.gov

Brigitte C. Widemann
Principal Investigator

Link to the current ClinicalTrials.gov record.
NLM Identifer NCT01660984

Note: Information about participating sites on pharmaceutical industry trials may be incomplete. Please visit the ClinicalTrials.gov record via the link above for more information about participating sites.