Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease

Early Phase 1TerminatedNCT01015300

Dartmouth-Hitchcock Medical Center1 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemangioblastomas Von Hippel Lindau Disease

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI)

Time frame: 24 months

Secondary Outcomes

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.