Clinical Trial: Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to Follow

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

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 Summary:

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 eligibl
Sponsor: M.D. Anderson Cancer Center

Current Primary Outcome: Safety of Sunitinib Administration in Participants With Von Hippel-Lindau Syndrome (VHL) [ Time Frame: 12 weeks ]

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.


Original Primary Outcome:

  • Results of degree & frequency of toxicity and degree of efficacy will be summarized (frequencies, rates, confidence intervals, etc).
  • In addition, changes in tumor size before and after treatment will be evaluated by repeated measures of variance.


Current Secondary Outcome: Number of VHL Lesion Complete + Partial Responses [ Time Frame: Baseline to 12 months (evaluations at 6 and 12 months) ]

Response of VHL lesions (number) evaluated using Response Evaluation Criteria in Solid Tumors (RECIST) of Complete Response (CR): Disappearance of all target lesions, and Partial Response (PR): At least a 30% decrease in the sum of longest diameter (LD) of target lesions, reference baseline sum LD. Progressive Disease (PD): 20% increase in LD sum and Stable Disease (SD): Insufficient shrinkage to qualify for PR nor increase to qualify for PD. Degree and timing of response in affected organs evaluated in order to determine organ specific kinetics of therapy.


Original Secondary Outcome:

Information By: M.D. Anderson Cancer Center

Dates:
Date Received: May 26, 2006
Date Started: May 2006
Date Completion:
Last Updated: February 23, 2012
Last Verified: February 2012