Vandetanib to Treat Children and Adolescents With Medullary Thyroid Cancer
- Medullary thyroid carcinoma (MTC) is common in people with a genetic disorder called multiple endocrine neoplasia (MEN).
- Vandetanib is an experimental drug that blocks a defective protein receptor (RET receptor) found on the surface of cancer cells in people with MEN. It is thought that this protein is a primary cause of MTC in people with MEN.
- To study the activity of Vandetanib in children and adolescents with MEN-related MTC by measuring the change in tumor size, in blood levels of proteins produced the tumor (calcitonin and CEA) and in tumor-related diarrhea.
- To determine the safety and tolerability of Vandetanib in children and adolescents.
- To study how the body handles Vandetanib in children and adolescents.
- To determine the effect of Vandetanib on the survival of children and adolescents with MTC.
-Children and adolescents 5 to 18 years of age with MTC whose tumor cannot be surgically removed or has grown back after treatment or has metastasized (spread beyond the thyroid gland).
- Patients take Vandetanib once a day in 28-day cycles. The first patients enrolled in the study are started on a low dose of Vandetanib to determine tolerability.
- Patients have periodic blood tests, electrocardiograms, and blood pressure measurements to look for side effects of Vandetanib.
- Blood tests and imaging scans (MRI, CT, bone and octreoscan) are done every 8 weeks for the first 32 weeks of treatment and then every 16 weeks for the duration of the treatment period.
- Patients who have tumor-related diarrhea keep a daily record of the number and consistency of bowel movements.
Medullary Thyroid Carcinoma
Multiple Endocrine Neoplasia Type 2A
Multiple Endocrine Neoplasia Type 2B
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase I/II Trial of Vandetanib (ZD6474, ZACTIMA) in Children and Adolescents With Hereditary Medullary Thyroid Carcinoma|
- Assess pharmacokinetics of vandetanib at steady state [ Time Frame: pre dose 1, 24 hrs after 1st dose, pre cycle 2, and post cycle 2 (this will include thorough eval) ] [ Designated as safety issue: No ]
- Assess safety and tolerance [ Time Frame: after 4 weeks of drug ] [ Designated as safety issue: Yes ]
- Assess the activity of vandetanib in children with MTC [ Time Frame: every odd cycle ] [ Designated as safety issue: No ]
- Assess a variety of research endpoints including expression of RET, EGFR, VEGFR and somatostatin, assess gene expression and screen for gains or losses of DNA sequences [ Time Frame: at time of biopsy ] [ Designated as safety issue: No ]
- Determine progression free survival and overall survival [ Time Frame: after every 2 cycles X4, then after every 4 cycles ] [ Designated as safety issue: No ]
|Study Start Date:||July 2007|
|Estimated Study Completion Date:||July 2020|
|Estimated Primary Completion Date:||April 2018 (Final data collection date for primary outcome measure)|
vandetanib daily X 28 days
once daily continuously (28 day cycles)
Show Detailed Description
Please refer to this study by its ClinicalTrials.gov identifier: NCT00514046
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Brigitte C Widemann, M.D.||National Cancer Institute (NCI)|