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.
Multiple Endocrine Neoplasia Type 2A
Multiple Endocrine Neoplasia Type 2B
Drug: ZACTIMA (Vandetanib)
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: 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|
- Objective response (complete or partial) as assessed by RECIST [ Designated as safety issue: No ]
- Change in tumor markers (calcitronin and CEA) [ Designated as safety issue: No ]
- Change in tumor-related diarrhea [ Designated as safety issue: No ]
- Safety [ Designated as safety issue: Yes ]
- Maximum tolerated dose [ Designated as safety issue: Yes ]
- Pharmacokinetics [ Designated as safety issue: No ]
- Toxicity [ Designated as safety issue: Yes ]
- Progression-free survival [ Designated as safety issue: No ]
- Overall survival [ Designated as safety issue: No ]
- Expression of RET, EGFR, VEGFR, and somatostatin receptor by IHC [ Designated as safety issue: No ]
- Tumor pharmacodynamics [ Designated as safety issue: No ]
|Study Start Date:||July 2007|
|Estimated Study Completion Date:||June 2015|
|Estimated Primary Completion Date:||June 2015 (Final data collection date for primary outcome measure)|
Drug: ZACTIMA (Vandetanib)
|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)|