Treatment of Von Hippel-Lindau (VHL)-Related Hemangioblastoma With PTK787/ZK 222584

Novartis

Status and phase

Completed

Phase 2

Conditions

Von Hippel-Lindau Disease

CNS Hemangioblastoma

Retinal Hemangioblastoma

Treatments

Drug: PTK787/ZK 222584

Study type

Interventional

Funder types

Industry

Identifiers

NCT00052013

CPTK787 0144

Details and patient eligibility

About

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).

Enrollment

11 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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