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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)

Phase 2
18 Years
Not Enrolling
Von Hippel-Lindau Disease, Central Nervous System Capillary Hemangioblastoma, Retinal Capillary Hemangioblastoma

Thank you

Trial Information

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)

Inclusion 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

- 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

- 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

- 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

- Patients with contraindication to MRI imaging

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Evaluate efficacy

Principal Investigator


Investigator Role:

Study Chair

Investigator Affiliation:



United States: Food and Drug Administration

Study ID:

CPTK787 0144



Start Date:

February 2003

Completion Date:

Related Keywords:

  • Von Hippel-Lindau Disease
  • Central Nervous System Capillary Hemangioblastoma
  • Retinal Capillary Hemangioblastoma
  • von Hippel-Lindau disease,
  • CNS,
  • hemangioblastoma,
  • retinal
  • Von Hippel-Lindau Disease
  • Hemangioblastoma



Duke University Medical Center Durham, North Carolina  27710
Dana Farber Cancer Institute Boston, Massachusetts  02115