Clinical trial NCT00330564
Conditions: Von Hippel-Lindau Syndrome Renal Cell Carcinoma Hemangioblastoma
Keywords: VHL Syndrome Von Hippel-Lindau Syndrome VHL Disease Kidney Retina Brain Spinal cord Pancreas Inner ear Cysts Tumors Sunitinib Malate SU011248 Sutent VHL Sunitinib Renal cell carcinoma Hemangioblastoma Endothelium
Sponsor: M.D. Anderson Cancer Center
Start date: 2006-05-01; Completion date: 2011-05-01
Enrollment: 15 participants; Allocation: NON_RANDOMIZED; Intervention model: SINGLE_GROUP

Brief summary:
The goal of this clinical research study is to learn if sunitinib malate (SU011248) can help to control VHL. The safety of this drug will also be studied.

Primary objectives:

* Evaluate safety of treatment with SU011248/sunitinib malate (50 mg daily dose for 4 weeks, then 2 weeks off) for 6 months in patients with Von Hippel-Lindau Syndrome (VHL) who have a measurable lesion undergoing surveillance

Secondary objectives:

* Evaluate efficacy of treatment with SU011248/sunitinib malate (50 mg daily dose for 4 weeks, then 2 weeks off) for 6 months in patients with VHL who have a measurable lesion undergoing surveillance

Correlative objectives:

* Evaluate quality of life of SU011248/sunitinib malate therapy in VHL patients
* Evaluate peripheral blood lymphocyte receptor phosphorylation in VHL patients taking SU011248/sunitinib malate (optional procedure)
* Correlate results of dynamic contrast-enhanced and diffusion weighted MRI and dynamic contrast enhanced CT with response and explore findings suggestive of surrogates of early response (optional procedure)

Detailed description:
Sunitinib malate is designed to block pathways that control important events such as the growth of blood vessels that are essential for the growth of cancer.

Before you can start treatment on this study, you will have what are called "screening tests." These tests will help the doctor decide if you are eligible to take part in this study. You will have standard scans to check the status of your disease, including computed tomography (CT) scans of the chest and abdomen (stomach area) and/or a magnetic resonance imaging (MRI) scan of the spine, if you have lesions in these areas. You will have an electrocardiogram (ECG -- a test that measures the electrical activity of the heart) and an echocardiogram or MUGA scan (echocardiogram/MUGA scan - a test to determine how well your heart is functioning by measuring its ability to pump blood). If the doctors know or suspect that VHL is affecting your eyes you will have an eye exam performed.

Your complete medical history will be recorded and you will have a physical exam, including measurement of your vital signs (blood pressure, heart rate, temperature, and breathing rate), height, and weight. You will be asked about any medications or treatments you are currently taking. Blood (about 2 teaspoons) will be drawn for routine tests. You will also be asked about your ability to perform daily activities. Women who are able to have children must have a negative blood pregnancy test.

You will be asked to complete 2 questionnaires that ask about your quality of life and your level of fatigue. It will take about 20 minutes to complete both questionnaires. The same questionnaires will then be completed 4-6 weeks later, and again at the end of treatment.

If you are found to be eligible to take part in this study, you will take sunitinib malate once a day, either with or without food. You will take the drug for 4 weeks in a row followed by 2 weeks of rest with no study drug. These 6 weeks are called a study "cycle".

Before beginning each new cycle, you will have a physical exam and your complete medical history will be recorded. Blood (about 1 teaspoon) will be drawn for routine tests. You will be asked about any drugs you have taken and any side effects you may have experienced. You will also be asked about your ability to perform daily activities.

At the end of Cycles 2 and 4, you will have CT or MRI scans to evaluate the status of your disease and eye exams may be repeated for those with known lesions on their eye(s).

You may receive treatment on this study for 24 weeks. However if you are showing benefit from the study drug, you may continue on study for an additional 24 weeks. (maximum total of 48 weeks) You will be taken off study if the disease gets worse or if intolerable side effects occur.

Once you stop treatment, you will have an end-of-study visit. At this visit, you will have a physical exam and blood (about 1 teaspoon) will be drawn for routine tests. You will be asked about any medications you have taken and any side effects you may have experienced. You will have CT or MRI scans to evaluate the status of your disease, as well as an eye exam if your eyes are affected by your disease. If you have completed at least 1 cycle of treatment on this study, and have had imaging scans in the past 28 days, you will skip the end of study evaluation and return for the 48 week follow-up visit.

You will have a follow-up visit about 48 weeks after your date of enrollment on this study. At this visit, you will have a physical exam and blood (about 1 teaspoon) will be drawn for routine tests. You will be asked about any medications you have taken and any side effects you may have experienced. You will have CT or MRI scans to evaluate the status of your disease. If your eyes are being affected by the disease, you will have a follow-up eye exam.

Once you complete the 48 week follow-up visit, you are considered off-study.

This is an investigational study. Sunitinib malate has been authorized by the FDA for research purposes only. About 28 patients will take part in this clinical research study. All will be enrolled at M. D. Anderson.

Arms: arm 1: 50 mg/day orally for 4 weeks
Interventions: intervention 1: SU011248
Intervention descriptions: intervention 1: 50 mg/day orally for 4 weeks, no treatment for 2 weeks (6 weeks = 1 cycle).

Protocol documents: