Clinical Trials Logo

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 June 3, 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 216
Est. completion date
Est. primary completion date
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 4 Months and older
Eligibility - INCLUSION CRITERIA FOR PATIENTS: - Patients must have histologically or cytologically confirmed MTC, confirmed by the Laboratory of Pathology, NCI OR Confirmation of MEN2A or MEN2B diagnosis, regardless of presence of MTC. - Performance Status: Ability to travel to the NIH and to undergo evaluations to be performed on this protocol. - Informed Consent: Ability of patient or their legal guardian (if the patient is <18 years old) to understand and willing to sign a written informed consent document. - Prior and current therapy: 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. EXCLUSION CRITERIA FOR PATIENTS: -In the opinion of the investigator the patient is not able to return for follow-up visits or obtain required follow-up studies. Inclusion Criteria for Parents or Primary Caregivers - Must be a parent or primary caregiver of a patient (< 21) who has a histologically or cytologically confirmed MTC or who have MEN2 (regardless of MTC status). - Ability to understand and be willing to sign a written informed consent document. EXCLUSION CRITERIA FOR PARENTS OR PRIMARY CAREGIVERS: None

Study Design


Locations

Country Name City State
United States National Institutes of Health Clinical Center Bethesda Maryland

Sponsors (1)

Lead Sponsor Collaborator
National Cancer Institute (NCI)

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Study growth rate of primary and metastatic tumor lesions Characterized features will be described and presented. 5 years
Primary Study molecular pathways altered in MTC Characterized features will be described and presented. 5 years
Secondary Describe the non-tumor related manifestations of MEN 2 Describe the non-tumor related manifestations of MEN 2 5 years
Secondary Investigate the psychosocial experiences Investigate the psychosocial experiences 5 years
See also
  Status Clinical Trial Phase
Active, not recruiting NCT03643055 - 18F-Fluorocholine PET/CT in Medullary Thyroid Cancer
Terminated NCT02709889 - Rovalpituzumab Tesirine in Delta-Like Protein 3-Expressing Advanced Solid Tumors Phase 1/Phase 2
Recruiting NCT06277180 - 68Ga-TCR-FAPI PET/CT Guided Precision Surgery for MTC N/A
Withdrawn NCT04760288 - A Study of Pralsetinib Versus Standard of Care (SOC) for Treatment of RET-Mutated Medullary Thyroid Cancer (MTC). Phase 3
Not yet recruiting NCT06079723 - A Detailed Look At What Patients Experience In Medullary Thyroid Cancer Clinical Study
Recruiting NCT01739634 - The Efficacy of CASAD in Patients With Diarrhea Related to Medullary Thyroid Cancer Phase 1/Phase 2
Recruiting NCT03157128 - A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001) Phase 1/Phase 2
Completed NCT02856347 - Prospective Study Evaluating the Medullary Thyroid Cancer Management's Care Using PET F-DOPA in Patients With a High Level of Postoperative Residual Thyrocalcitonin N/A
Completed NCT03037385 - Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors Phase 1/Phase 2
Active, not recruiting NCT04280081 - A Study of Selpercatinib (LY3527723) in Participants With Advanced Solid Tumors Including RET Fusion-positive Solid Tumors, Medullary Thyroid Cancer and Other Tumors With RET Activation Phase 2
Not yet recruiting NCT06292988 - Predictive Factors for Medullary Thyroid Cancer Aggressiveness
Completed NCT01757470 - Vandetanib Risk Minimisation Effectiveness N/A
Recruiting NCT05830500 - Study of Anlotinib in Patients With Advanced Medullary Thyroid Carcinoma Phase 4
Recruiting NCT03899792 - A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors Phase 1/Phase 2
Withdrawn NCT03838692 - Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer Phase 2
Terminated NCT02363647 - Personalized Cancer Therapy for Patients With Metastatic Medullary Thyroid or Metastatic Colon Cancer N/A
Terminated NCT00582712 - An Initial Study of Lithium in Patients With Medullary Thyroid Cancer Phase 2
Recruiting NCT06141369 - Treatment of Advanced Endocrine Tumor With Iindividualized mRNA Neoantigen Vaccine (mRNA-0523-L001) N/A
Completed NCT06243965 - Is Desmoplastic Stromal Reaction Useful to Modulate Lymph Node Dissection in Sporadic Medullary Thyroid Carcinoma?
Active, not recruiting NCT01298323 - Study to Determine if Contacting Patients With MTC More Frequently Results in Earlier Detection and Treatment of Signs and Symptoms of AEs and Thus a Decrease in the Percentage of Time Patients Experience AEs During First 12 Months on Vandetanib Treatment Phase 3