Natural History of Medullary Thyroid Cancer to Inform Advanced Disease Management

Study Overview
=================
Brief Summary
-----------------
This study utilizes a multi-institutional registry to describe the natural history of medullary thyroid cancer that has spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) in understanding disease management. The goal of this study is to learn about how medullary thyroid cancer develops and progresses.

Detailed Description
-----------------
PRIMARY OBJECTIVES: I. To describe the natural history of medullary thyroid cancer (MTC) using a comprehensive battery of demographic, clinical, pathologic, and genotypic variables collected in our existing multi-institutional patient registry. Ia. To describe the demographic, clinical, pathologic, and genotypic variables associated with each phase of disease. Ib. To identify those variables which are predictors of progression to the most advanced phases of disease that require small molecule therapy, including commercially approved and experimental agents. II. To characterize patients' experience with the different phases of MTC through a systematic evaluation of patient-reported outcomes (PROs). IIa. To describe clinical, psychosocial, economic, and physical well-being variables and the subsequent impact on quality of life associated with each phase of disease. IIb. To evaluate longitudinal changes in clinical, psychosocial, economic, and physical well-being variables between and within all phases of MTC, and the subsequent impact they have on quality of life. III. To evaluate the association of selected biometric and patient-reported outcomes on adherence, change, and discontinuation of approved targeted therapies and drugs being tested within clinical trial. OUTLINE: AIM I & II: Patients complete questionnaires over 10-40 minutes 2-4 times per year about health and how finances and quality of life effect experience with disease. AIM III: Patients complete a questionnaire over 2 minutes and undergo blood pressure measurements every day for up to 12 weeks. The questionnaire data gets paired with the collected medical record history that is maintained in the Registry.

Official Title
-----------------
Utilization of the Natural History of Medullary Thyroid Carcinoma to Inform Advanced Disease Management

Conditions
-----------------
Advanced Thyroid Gland Medullary Carcinoma, Stage III Thyroid Gland Medullary Carcinoma AJCC v8, Stage IV Thyroid Gland Medullary Carcinoma AJCC v8, Stage IVA Thyroid Gland Medullary Carcinoma AJCC v8, Stage IVB Thyroid Gland Medullary Carcinoma AJCC v8, Stage IVC Thyroid Gland Medullary Carcinoma AJCC v8

Intervention / Treatment
-----------------
* Other: Blood Pressure Measurement
* Other: Questionnaire Administration


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Aims 1 and 2: A diagnosis of MTC Aims 1 and 2: A registrant in the Medullary Thyroid Cancer Registry (MTCR). Patients of all disease phases will be eligible for enrollment Aim 3: A diagnosis of advanced phase (red or gray) MTC as determined by clinical team Aim 3: A current registrant in the MTCR

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Observational (questionnaire, blood pressure)<br>AIM I & II: Patients complete questionnaires over 10-40 minutes 2-4 times per year about health and how finances and quality of life effect experience with disease. AIM III: Patients complete a questionnaire over 2 minutes and undergo blood pressure measurements every day for up to 12 weeks. | Other: Blood Pressure Measurement<br>* Undergo blood pressure measurement<br>Other: Questionnaire Administration<br>* Complete questionnaires<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Natural history of medullary thyroid cancer (MTC) | Will describe the natural history of MTC using comprehensive battery of demographic, clinical, pathologic, and genotypic variables collected in existing multi-institutional patient registry. | Up to 4 years | 
| Patients' experience with the different phases of MTC | Will characterize patients' experience with the different phases of MTC through a systematic evaluation of patient-reported outcomes (PROs). | Up to 4 years | 
| Association of selected biometric and patient-reported outcomes | Will evaluate the association of selected biometric and patient-reported outcomes on adherence, change, and discontinuation of approved targeted therapies and drugs being tested within clinical trial. | Up to 4 years |