Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease
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ClinicalTrials.gov Identifier: NCT01015300
Recruitment Status :
(Study terminated due to low accrual.)
Von Hippel-Lindau (VHL) disease is an inherited syndrome manifested by a variety of benign and malignant tumors. Hemangioblastomas are the most common lesion associated with VHL disease affecting 60-84% of patients with a mean age at diagnosis of 29 years. Standard treatment for this disease is by surgery or radiotherapy. No approved systemic therapy yet exists. Patients with VHL have an increased growth factor production, specifically vascular endothelial growth factor (VEGF), resulting in angiogenesis (growth of blood vessels). Studies show that Bevacizumab inhibits the growth of VEGF protein and will block the VEGF-driven angiogenesis and result in stabilization and regression of hemangioblastomas in VHL disease patients. The dose of bevacizumab will be 10 mg/kg every two weeks for up to 6 months.
Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI) [ Time Frame: 24 months ]
Secondary Outcome Measures :
Changes in VEGF with bevacizumab treatment assist in the predication of radiographic response. Products of the HIF-1A synthesis pathway: plasma VEGF, PDGF, TGF-a and erythropoietin. [ Time Frame: 24 months ]
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Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
One or more CNS hemangioblastomas not amendable to surgical resection or recurrent post resection
Confirmed diagnosis of von-Hippel-Lindau disease
No prior treatment with VEGF inhibitors
Index hemangioblastomas lesion at least 5mm on MRI
No major bleeding event from hemangioblastoma within 90 days
KPS > or equal to 60%
Age > or equal to 18 years
Prior treatment with VEGF inhibitors
Major bleeding event from hemangioblastoma within 90 days
Inability to comply with study and/or follow up procedures
Life expectancy of less than 12 weeks
Current or recent (within 4 weeks of the first infusion of this study) participation in an experimental drug study other than a Genentech sponsored bevacizumab cancer study
Active malignancy will be permissible if treating physician deems that concurrent administration of bevacizumab is not contraindicated and that the patient would be able to complete with the other parameters of the protocol