Von Hippel-Lindau DiseasePhase 2NCT00052013
Treatment of Von Hippel-Lindau (VHL)-Related Hemangioblastoma With PTK787/ZK 222584
A Phase 2 study testing PTK787/ZK 222584 in people with Von Hippel-Lindau Disease and CNS Hemangioblastoma, run by Novartis Pharmaceuticals at 2 sites in United States.
Trial summary
The purpose of this study is to determine whether PTK787/ZK 222584 is effective in treating hemangioblastoma of the brain and/or retina in patients with von Hippel-Lindau disease. The study will also assess safety and tolerability of PTK787/ZK 222584, and changes in markers of angiogenesis (new blood vessel growth). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Von Hippel-Lindau Disease, CNS Hemangioblastoma, Retinal Hemangioblastoma
- What is being tested?
- PTK787/ZK 222584
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 2 sitesUnited States
- Study dates
- Feb 2003to Jun 2006
- Phase
- Phase 2
- 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
- Confirmed diagnosis of VHL disease
- One or more measurable CNS hemangioblastoma, if symptomatic, must be refractory or not amenable to standard therapy, if asymptomatic, must have had at least one prior neurosurgical treatment/ procedure; and/or: at least one untreatable or treatment-refractory retinal hemangioblastoma that is causing impaired visual function
- Karnofsky Performance Status >=60
- Life expectancy > 3 months
- Able to sign informed consent
- Adequate hematologic status, liver and kidney function
Who cannot join
- Patients with other VHL-related tumors requiring or amenable to standard treatment
- Severe or uncontrolled concurrent illnesses that could compromise participation in the study
- Total urinary protein in 24 hour collection > 500 mg
- Pregnant or breast feeding females, adults of reproductive potential not using effective contraception (hormonal methods not considered effective due to possible decreased effectiveness secondary to drug interaction with PTK787). Women of childbearing potential must have negative serum pregnancy test prior to initiation of treatment.
- Acute or chronic liver disease
- Diagnosis of HIV infection
Show all 13Show fewer
- GI function that may alter absorption of PTK787
- Patients taking coumadin (warfarin sodium)
- Prior therapies (investigational drugs, chemotherapy) within 4 weeks prior to study entry
- Prior therapies (biologic, hormonal, immunotherapy, radiation therapy, surgery) within two weeks prior to study entry.
- Patients unwilling or unable to comply with protocol requirements
- Patients with concurrent, non VHL-related malignancies other than non-melanoma skin cancer
- Patients with contraindication to MRI imaging
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
Duke University Medical Center
Durham, North Carolina, 27710
About this study
Official title: A Phase II Open-Label Study of Oral, Continuous, Once Daily PTK787/ZK 222584 in Patients With Von Hippel-Lindau Disease (VHL) and Hemangioblastoma (HB)
The purpose of this study is to determine whether PTK787/ZK 222584 is effective in treating hemangioblastoma of the brain and/or retina in patients with von Hippel-Lindau disease. The study will also assess safety and tolerability of PTK787/ZK 222584, and changes in markers of angiogenesis (new blood vessel growth).
- Being tested
- PTK787/ZK 222584
- Keywords
- von Hippel-Lindau disease,, CNS,, hemangioblastoma,, retinal
- Medical subject headings
- von Hippel-Lindau Disease, Hemangioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTK787/ZK 222584Drug
| Study group | Role | What they receive |
|---|---|---|
| PTK787/ZK 222584 | Experimental | Not described Receives: Drug: PTK787/ZK 222584 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate efficacy
Secondary outcomes (3)
Evaluate changes in dynamic contrast enhanced magnetic resonance imaging
To explore the correlation of the pharmacokinetics
To assess changes in surrogate markers of angiogenesis
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2003 (estimated)
- Study startFeb 2003
- Primary completionJun 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Hippel-Lindau DiseaseStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Novartis, Study chair, Novartis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00052013
- Sponsor's protocol ID
- CPTK787 0144
- Status last verified
- Feb 2011
- First submitted
- 21 Jan 2003
- Last updated
- 6 Mar 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Open-Label Study of Oral, Continuous, Once Daily PTK787/ZK 222584 in Patients With Von Hippel-Lindau Disease (VHL) and Hemangioblastoma (HB)
- ClinicalTrials.gov ID
- NCT00052013
- Acronym / sponsor ID
- CPTK787 0144
- Keywords
- von Hippel-Lindau disease,, CNS,, hemangioblastoma,, retinal
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2003
- Primary completion
- Jun 2006 (actual)
- First posted
- 22 Jan 2003 (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.
The purpose of this study is to determine whether PTK787/ZK 222584 is effective in treating hemangioblastoma of the brain and/or retina in patients with von Hippel-Lindau disease. The study will also assess safety and tolerability of PTK787/ZK 222584, and changes in markers of angiogenesis (new blood vessel growth).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PTK787/ZK 222584 | Experimental | Drug: PTK787/ZK 222584 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Confirmed diagnosis of VHL disease
- One or more measurable CNS hemangioblastoma, if symptomatic, must be refractory or not amenable to standard therapy, if asymptomatic, must have had at least one prior neurosurgical treatment/ procedure; and/or: at least one untreatable or treatment-refractory retinal hemangioblastoma that is causing impaired visual function
- Karnofsky Performance Status >=60
- Life expectancy > 3 months
- Able to sign informed consent
Key exclusion criteria (13 listed)
- Patients with other VHL-related tumors requiring or amenable to standard treatment
- Severe or uncontrolled concurrent illnesses that could compromise participation in the study
- Total urinary protein in 24 hour collection > 500 mg
- Pregnant or breast feeding females, adults of reproductive potential not using effective contraception (hormonal methods not considered effective due to possible decreased effectiveness secondary to drug interaction with PTK787). Women of childbearing potential must have negative serum pregnancy test prior to initiation of treatment.
- Acute or chronic liver disease
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 |
|---|---|---|
| PTK787/ZK 222584 | Drug | PTK787/ZK 222584 |
Primary outcome
Evaluate efficacy
3 secondary outcomes
Evaluate changes in dynamic contrast enhanced magnetic resonance imaging
To explore the correlation of the pharmacokinetics
To assess changes in surrogate markers of angiogenesis
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2017.
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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.
Novartis study chair
Novartis
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PTK787/ZK 222584 in people with Von Hippel-Lindau Disease and CNS Hemangioblastoma, run by Novartis Pharmaceuticals at 2 sites 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 6 inclusion and 13 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: finished, as of the sponsor's last update on 6 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Evaluate efficacy. 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 6 Mar 2017.