HemangioblastomasEarly Phase 1NCT01015300
Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease
A Early Phase 1 study testing Avastin in people with Hemangioblastomas and Von Hippel Lindau Disease, run by Dartmouth-Hitchcock Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemangioblastomas, Von Hippel Lindau Disease
- What is being tested?
- Avastin
- Type of treatment
- Injection or infusion
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2009to Apr 2012
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
About this study
Official title: D0904 - A Pilot Study of Bevacizumab (Avastin) in Patients With Unresectable or Recurrent Hemangioblastoma From Von Hippel-Lindau Disease.
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
- Being tested
- Avastin
- Keywords
- Hemangioblastoma, Von Hippel Lindau, VHL, lesion, EGFR
- Medical subject headings
- Hemangioblastoma, von Hippel-Lindau Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvastinDrug
Patients will receive Bevacizumab (Avastin) 10mg/kg IV every two weeks for 6 months
What is being measured
The primary outcome is the main question the study is designed to answer.
Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI)
Measured: 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.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2009 (estimated)
- Study startDec 2009
- Primary completionOct 2011
- Study completionApr 2012
Why it stopped: Study terminated due to low accrual.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemangioblastomasStudies of the same condition, open and under way.
- More trials run by Dartmouth-Hitchcock Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dartmouth-Hitchcock Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- J Marc Pipas, MD, Principal investigator, Dartmouth-Hitchcock Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01015300
- Sponsor's protocol ID
- D0904
- Status last verified
- May 2012
- First submitted
- 16 Nov 2009
- Last updated
- 11 May 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- D0904 - A Pilot Study of Bevacizumab (Avastin) in Patients With Unresectable or Recurrent Hemangioblastoma From Von Hippel-Lindau Disease.
- ClinicalTrials.gov ID
- NCT01015300
- Acronym / sponsor ID
- D0904
- Conditions
- Hemangioblastomas, Von Hippel Lindau Disease
- Keywords
- Hemangioblastoma, Von Hippel Lindau, VHL, lesion, EGFR
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study terminated due to low accrual.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Dartmouth-Hitchcock Medical CenterUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Dec 2009
- Primary completion
- Oct 2011 (actual)
- Study completion
- Apr 2012 (actual)
- First posted
- 18 Nov 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AvastinPatients will receive Bevacizumab (Avastin) 10mg/kg IV every two weeks for 6 months | Drug | – |
Primary outcome
Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI)
Time frame: 24 months
1 secondary outcome
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
Results and safety
The study was stopped early (reason given: Study terminated due to low accrual.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
J Marc Pipas, MD principal investigator
Dartmouth-Hitchcock Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Avastin in people with Hemangioblastomas and Von Hippel Lindau Disease, run by Dartmouth-Hitchcock Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 May 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Study terminated due to low accrual.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Radiographic response in the size of the hemangioblastoma on magnetic resonance imaging (MRI) (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 May 2012.