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Investigator(s):
Frank Balis, M.D. Principal Investigator Phone: 301-496-0085 balisf@mail.nih.gov
Referral Contact(s):
Pediatric Oncology  Phone: 1-877-624-4878 (Toll free)

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Primary Eligibility:

• Histologically confirmed unresectable, recurrent, or metastatic hereditary (MEN 2A or 2B) medullary thyroid carcinoma
• Documented characteristic germline mutation in the RET proto-oncogene obtained from the referring institution
• Measurable disease, defined by Response Evaluation Criteria in Solid Tumors (RECIST) and superficial, palpable lymph nodes
• No pheochromocytoma as evidenced by elevated plasma-free metanephrines
• Prior therapy
• Must have recovered to grade 1 from toxic effects of prior therapy
• ≥ 4 weeks since surgery (incisions must be healed)
• ≥ 4 weeks since last fraction of external beam radiation
• ≥ 28 days since cytotoxic chemotherapy
• ≥ 7 days since biological therapy, immunomodulary agents, vaccines, differentiating agents to treat cancer
• ≥ 30 days since monoclonal antibodies
• ≥ 30 days since the last dose of investigational agents
• ≥ 72 hours since prior short-acting colony-stimulating factors (e.g., filgrastim [G-CSF] or sargramostim [GM-CSF]) and ≥ 7 days since PEG-filgrastim
• ≥ 5 years of age and ≤ 18 years of age
• Lansky (≤ 10 years of age) or Karnofsky (> 10 years of age) performance status 50%–100%
• ANC ≥ 1,500/μL
• Platelet count ≥ 100,000/μL
• PT and PTT ≤ 1.5 x upper limit of normal (ULN)
• Bilirubin ≤ 1.5 x ULN
• AST and ALT ≤ 2.5 x ULN (5 x ULN in patients with hepatic metastasis)
• Creatinine clearance ≥ 60 mL/min/1.73m² OR normal age-adjusted serum creatinine
• Potassium < 3.5 mmol/L or serum calcium or magnesium < LLN; electrolytes may be corrected with supplements
• No history of arrhythmia that is symptomatic or requires treatment (except for controlled atrial fibrillation)
• No QTc prolongation (congenitally prolonged QTc, first-degree relative with sudden death < 40 yrs old, QTc > 480 msec); patient experienced prolonged QTC with other medications, or receiving medications known to prolong QTc within 14 days (28 days for levomethadyl)
• Diastolic blood pressure ≤ 95% for age; no anti-hypertensive therapy
• Must be able to take oral medications
• No uncontrolled or severe systemic illness
• Not pregnant or nursing; fertile patients must use effective contraception during the study and for 2 months after the completion of study therapy
• Willing to use birth control during study and until 2 months after the last dose

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Treatment Plan:

This study is being conducted at the NIH Clinical Center and includes a limited dose escalation portion (phase I). For the phase I portion, patients are stratified according to age (13–18 vs 5–12) and older patients are studied before younger patients. All patients are monitored for response.

• Patients receive oral vandetanib once daily on Days 1–28 (one cycle)
• Treatment cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity
• Patients are monitored for toxicity (QTc prolongation, high blood pressure, fatigue, nausea/vomiting, diarrhea, rash) and response (OctreoScan, CT, MRI, bone scans, CEA levels, calcitonin levels, severity of tumor-related diarrhea)
• Blood is collected periodically for research studies
• After completion of study treatment, patients are followed periodically

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Additional Information:

• This trial will be conducted at the NIH Clinical Center in Bethesda, MD. It is open to patients who meet the eligibility requirements, regardless of where they live in the United States.
• There is no charge for medical care received at NIH Clinical Center.
• FAQs about this study - provides information for patients about the trial such as frequency and duration of visits, costs, how to enroll, treatment plan.
• PDQ (Physicians Data Query) - provides additional details about this study for health care providers.

Reviewed: 11/10/08
Updated: 7/31/08