Know Cancer

forgot password

A Phase 2 Study of SU011248 (Sunitinib Malate) in Von Hippel-Lindau Syndrome

Phase 2
18 Years
Not Enrolling
Von Hippel-Lindau Syndrome, Renal Cell Carcinoma, Hemangioblastoma

Thank you

Trial Information

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
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

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.

Inclusion Criteria:

- Patients must have genetically confirmed Von Hippel-Lindau (VHL) disease.

- At least one measurable VHL-related lesion, which is undergoing surveillance, and
patient is not at immediate risk of needing intervention for this or other lesions.
Biopsy is not required given the known natural history in the setting of a positive
genetic test. (1) Renal: solid mass suspicious for Renal Cell Carcinoma (RCC) >/= 1
cm or cystic mass >/= 1 cm; (2) Pancreas: Solid mass >/= 1cm & < 3cm suspicious for
neuroendocrine tumor; (3) Brain: asymptomatic hemangioblastoma > 5mm; (4) Spine:
asymptomatic hemangioblastoma, > 1cm; (5) Eye: asymptomatic peripapillary and/or
macular hemangioblastoma, any size.

- Allowable prior therapy: (1) Patients having undergone prior therapy for VHL lesions
may enroll as long as other criteria are met. Previously radiated lesions may not be
considered as target lesions unless they demonstrate unequivocal evidence of growth;
(2) Major surgery, chemotherapy or radiation therapy completed >4 weeks prior to
starting the study treatment.

- Age >/= 18 years. Because no dosing or adverse event data are currently available on
the use of SU011248/sunitinib malate in patients <18 years of age, children are
excluded from this study but will be eligible for future pediatric single-agent
trials, if applicable.

- Eastern Cooperative Oncology Group (ECOG) performance status
- Patients must have normal organ and marrow function as defined: (1) serum aspartate
transaminase ([AST]; serum glutamic oxaloacetic transaminase [SGOT]) and serum
alanine transaminase ([ALT]; serum glutamic pyruvic transaminase [SGPT]) local laboratory upper limit of normal (ULN), or AST and ALT function abnormalities are due to underlying malignancy; (2) Total serum bilirubin
/= 1500mcL; (4) Platelets >/=
100,000 mcL; (5) Hemoglobin >/= 9.0 g/dL; (6) Serum creatinine < 1.5 x UL.

- Eligibility of patients receiving any medications or substances known to affect or
with the potential to affect the activity or pharmacokinetics of SU011248/sunitinib
malate as listed below will be determined following review of their case by the
Principal Investigators. If possible, efforts will be made to switch motivated
patients to other medications, otherwise patients will be excluded: (1) Ketoconazole,
(2) Theophylline, (3) Phenobarbital, (4) Coumadin at therapeutic doses (low dose
Coumadin up to 2 mg po daily for thromboprophylaxis is allowed).

- Ability to understand and the willingness to sign a written informed consent

Exclusion Criteria:

- Patients who have had chemotherapy or radiotherapy within 4 weeks prior to entering
the study or those who have not recovered from adverse events due to agents
administered more than 4 weeks earlier.

- Patients may not be receiving any other investigational agents.

- Patients with any metastatic disease of any kind.

- History of allergic reactions attributed to compounds of similar chemical or biologic
composition to SU011248/sunitinib malate.

- National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events
(CTCAE) grade 3 hemorrhage within 4 weeks of starting the study treatment.

- History of or known brain metastases, spinal cord compression, or carcinomatous
meningitis, or evidence of symptomatic brain or leptomeningeal disease on screening
Computer tomography (CT) or Magnetic Resonance Imaging (MRI) scan.

- Any of the following within the 6 months prior to study drug administration:
myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass
graft, symptomatic congestive heart failure, cerebrovascular accident or transient
ischemic attack, or pulmonary embolism.

- Ongoing cardiac dysrhythmias of NCI CTCAE grade >/= 2.

- Prolonged QTc interval on baseline electrocardiogram (ECG or EKG)>470ms.

- Hypertension that cannot be controlled by medications (>140/90 mm Hg despite optimal
medical therapy).

- Pre-existing thyroid abnormality with thyroid function that cannot be maintained in
the normal range with medication.

- Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situations that would limit compliance with
study requirements.

- Pregnant women are excluded from this study because SU011248/sunitinib malate has the
potential for teratogenic or abortifacient effects. Because there is an unknown but
potential risk for adverse events in nursing infants secondary to treatment of the
mother with SU011248/sunitinib malate, breastfeeding should be discontinued if the
mother is treated with SU011248/sunitinib malate.

- Known HIV-positive patients taking combination antiretroviral therapy are ineligible
because of the potential for pharmacokinetic interactions with SU011248/sunitinib
malate. Appropriate studies will be undertaken in patients receiving combination
antiretroviral therapy when indicated.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety of Sunitinib Administration in Participants With Von Hippel-Lindau Syndrome (VHL)

Outcome Description:

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.

Outcome Time Frame:

12 weeks

Safety Issue:


Principal Investigator

Eric Jonasch, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

May 2006

Completion Date:

May 2011

Related Keywords:

  • Von Hippel-lindau Syndrome
  • Renal Cell Carcinoma
  • Hemangioblastoma
  • 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
  • Carcinoma
  • Carcinoma, Renal Cell
  • Von Hippel-Lindau Disease
  • Hemangioblastoma



UT MD Anderson Cancer Center Houston, Texas  77030