Hemangioblastomas, Von Hippel Lindau Disease
Conditions
Keywords
Hemangioblastoma, Von Hippel Lindau, VHL, lesion, EGFR
Brief summary
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.
Interventions
DRUGAvastin
Patients will receive Bevacizumab (Avastin) 10mg/kg IV every two weeks for 6 months
Sponsors
Genentech, Inc.
Dartmouth-Hitchcock Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI) | 24 months |
Secondary
| Measure | Time frame |
|---|---|
| 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. | 24 months |
Countries
United States
Outcome results
None listed