Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease
Primary Purpose
Hemangioblastomas, Von Hippel Lindau Disease
Status
Terminated
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Avastin
Sponsored by
About this trial
This is an interventional treatment trial for Hemangioblastomas focused on measuring Hemangioblastoma, Von Hippel Lindau, VHL, lesion, EGFR
Eligibility Criteria
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
Sites / Locations
- Dartmouth Hitchcock Medical Center
Outcomes
Primary Outcome Measures
Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI)
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.
Full Information
NCT ID
NCT01015300
First Posted
November 16, 2009
Last Updated
May 9, 2012
Sponsor
Dartmouth-Hitchcock Medical Center
Collaborators
Genentech, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT01015300
Brief Title
Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease
Official Title
D0904 - A Pilot Study of Bevacizumab (Avastin) in Patients With Unresectable or Recurrent Hemangioblastoma From Von Hippel-Lindau Disease.
Study Type
Interventional
2. Study Status
Record Verification Date
May 2012
Overall Recruitment Status
Terminated
Why Stopped
Study terminated due to low accrual.
Study Start Date
December 2009 (undefined)
Primary Completion Date
October 2011 (Actual)
Study Completion Date
April 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Dartmouth-Hitchcock Medical Center
Collaborators
Genentech, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemangioblastomas, Von Hippel Lindau Disease
Keywords
Hemangioblastoma, Von Hippel Lindau, VHL, lesion, EGFR
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Avastin
Intervention Description
Patients will receive Bevacizumab (Avastin) 10mg/kg IV every two weeks for 6 months
Primary Outcome Measure Information:
Title
Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI)
Time Frame
24 months
Secondary Outcome Measure Information:
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
J Marc Pipas, MD
Organizational Affiliation
Dartmouth-Hitchcock Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Dartmouth Hitchcock Medical Center
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease
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