Clinical trial NCT00052013: Treatment of Von Hippel-Lindau (VHL)-Related Hemangioblastoma With PTK787/ZK 222584
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Von Hippel-Lindau Disease
CNS Hemangioblastoma
Retinal Hemangioblastoma
Keywords: von Hippel-Lindau disease,
CNS,
hemangioblastoma,
retinal
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2003-01-22
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Evaluate efficacy
Secondary outcomes:
measure: Evaluate changes in dynamic contrast enhanced magnetic resonance imaging
measure: To explore the correlation of the pharmacokinetics
measure: To assess changes in surrogate markers of angiogenesis
Other outcomes:
Locations:
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
Study officials:
affiliation: Novartis
name: Novartis
role: STUDY_CHAIR
References: