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

This study has been completed.
Information provided by:
Novartis Identifier:
First received: January 21, 2003
Last updated: March 2, 2017
Last verified: February 2011
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).

Condition Intervention Phase
Von Hippel-Lindau Disease CNS Hemangioblastoma Retinal Hemangioblastoma Drug: PTK787/ZK 222584 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Evaluate efficacy

Secondary Outcome Measures:
  • Evaluate changes in dynamic contrast enhanced magnetic resonance imaging
  • To explore the correlation of the pharmacokinetics
  • To assess changes in surrogate markers of angiogenesis

Enrollment: 11
Study Start Date: February 2003
Primary Completion Date: June 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PTK787/ZK 222584 Drug: PTK787/ZK 222584


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts 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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00052013

United States, Massachusetts
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Chair: Novartis Novartis
  More Information

Additional Information:
Responsible Party: External Affairs, Novartis Pharmaceuticals Identifier: NCT00052013     History of Changes
Other Study ID Numbers: CPTK787 0144
Study First Received: January 21, 2003
Last Updated: March 2, 2017

Keywords provided by Novartis:
von Hippel-Lindau disease,

Additional relevant MeSH terms:
Von Hippel-Lindau Disease
Neurocutaneous Syndromes
Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Hemangioma, Capillary
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action processed this record on September 25, 2017