Longitudinal Assessment and Natural History of Younger Patients with Multiple Endocrine Neoplasia 2A or 2B with or without Medullary Thyroid Cancer and Their Caregivers
- Patients must have histologically or cytologically confirmed MTC, confirmed by the Laboratory of Pathology, National Cancer Institute (NCI) OR
- Confirmation of MEN2A or MEN2B diagnosis, regardless of presence of MTC
- Ability to travel to the National Institutes of Health (NIH) and to undergo evaluations to be performed on this protocol
- 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
- 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 MEN 2 related manifestation(s) 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
- 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
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 MEN2 with or without MTC, irrespective of whether they are receiving tumor directed treatment.
III. Study molecular pathways altered in MEN2 with or without MTC and their relationship to the patients’ clinical course and response to targeted therapies.
I. Describe the non-tumor related manifestations of MEN 2 including skeletal, respiratory, 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 MEN2 and their caregivers, and assess how these change over time.
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.
Trial Phase Phase NA
Trial Type Ancillary-correlative
NCI - Center for Cancer Research
John W. Glod
- Primary ID 12-C-0178
- Secondary IDs NCI-2015-01255, 321080, P12989
- Clinicaltrials.gov ID NCT01660984