A Phase 2 Study of SU011248 (Sunitinib Malate) in Von Hippel-Lindau Syndrome
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
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
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.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Safety of Sunitinib Administration in Participants With Von Hippel-Lindau Syndrome (VHL)
Safety evaluation = Number of participants with treatment terminating toxicity using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 3.0. Early stopping rules applied when treatment terminating toxicity occurred in the first 6 week cycle. Recurring grade 3 toxicity requires dose reduction, with no more than 2 dose reductions permitted. If no improvement after 4 weeks, patient is taken off drug and off study, and the event recorded as treatment terminating toxicity.
Eric Jonasch, MD
M.D. Anderson Cancer Center
United States: Food and Drug Administration
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