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

NCT01015300 | Terminated Early Phase 1 DMC

• 1 other identifier: D0904
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemangioblastomas; Von Hippel Lindau Disease

Keywords

Hemangioblastoma; Von Hippel Lindau; VHL; lesion; EGFR

Outcome Measures

Primary Outcomes (1)

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

  24 months

Secondary Outcomes (1)

• 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

Interventions

Avastin DRUG

Patients will receive Bevacizumab (Avastin) 10mg/kg IV every two weeks for 6 months

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Dartmouth-Hitchcock Medical Center: lead
• Genentech, Inc.: collaborator

Study Sites (1)

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

MeSH Terms

Conditions

Hemangioblastoma; von Hippel-Lindau Disease

Interventions

Bevacizumab

Condition Hierarchy (Ancestors)

Hemangioma, Capillary; Hemangioma; Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Neoplasms; Neurocutaneous Syndromes; Nervous System Diseases; Angiomatosis; Vascular Diseases; Cardiovascular Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• J Marc Pipas, MD

  Dartmouth-Hitchcock Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2009
• First Posted: November 18, 2009
• Study Start: December 1, 2009
• Primary Completion: October 1, 2011
• Study Completion: April 1, 2012
• Last Updated: May 11, 2012

Record last verified: 2012-05

Locations

US (1)