CNS Hemangioblastoma, Retinal Hemangioblastoma, Von Hippel-Lindau Disease
Conditions
Keywords
von Hippel-Lindau disease,, CNS,, hemangioblastoma,, retinal
Brief 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).
Interventions
DRUGPTK787/ZK 222584
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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 * 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Evaluate efficacy | — |
Secondary
| Measure | Time frame |
|---|---|
| Evaluate changes in dynamic contrast enhanced magnetic resonance imaging | — |
| To explore the correlation of the pharmacokinetics | — |
| To assess changes in surrogate markers of angiogenesis | — |
Countries
United States
Outcome results
None listed