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

Sponsor

Dartmouth-Hitchcock Medical Center (Other)

Overall Status

Terminated

CT.gov ID

NCT01015300

Collaborator

Genentech, Inc. (Industry)

Enrollment

Location

Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Hemangioblastomas Von Hippel Lindau Disease	Drug: Avastin	Early Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

1 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

D0904 - A Pilot Study of Bevacizumab (Avastin) in Patients With Unresectable or Recurrent Hemangioblastoma From Von Hippel-Lindau Disease.

Study Start Date :

Dec 1, 2009

Actual Primary Completion Date :

Oct 1, 2011

Actual Study Completion Date :

Apr 1, 2012

Outcome Measures

Primary Outcome Measures

Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI) [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. [24 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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