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

Related Clinical Trial
Propranolol and Von Hippel-Lindau Disease Retrospective Case Series of Trans-scleral Cryotherapy for Retinal Hemangioblastoma 68Ga-DOTA-TOC PET/CT in Imaging Participants With Neuroendocrine Tumors Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy Study of Brain and Spinal Cord Tumor Growth and Cyst Development in Patients With Von Hippel Lindau Disease Assessment of Residual VHL Function in Tumors – Can it Predict the Patients’ Individual Course of Disease? Phase II Study of Vandetanib in Individuals With Kidney Cancer A Phase 2 Study of PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma Drivers of Hypoxia-induced Angiogenesis in Tumor Development Evaluation of 68Gallium-DOTATATE PET/CT for Detecting Neuroendocrine Tumors MyVHL: Patient Natural History Study Metabolic Mapping to Measure Retinal Metabolism Use of Tracking Devices to Locate Abnormalities During Invasive Procedures National Eye Institute Biorepository for Retinal Diseases Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to Follow Pazopanib in Von Hippel-Lindau (VHL) Syndrome 17AAG to Treat Kidney Tumors in Von Hippel-Lindau Disease Effect of Vorinostat on Nervous System Hemangioblastomas in Von Hippel-Lindau Disease (Missense Mutation Only) A Phase I/II Trial for Intravitreous Treatment of Severe Ocular Von Hippel-Lindau Disease Using a Combination of the PDGF Antagonist E10030 and the VEGF Antagonist Ranibizumab Ranibizumab Injections to Treat Retinal Tumors in Patients With Von Hippel-Lindau Syndrome Screening for Endolymphatic Sac Tumours (ELSTs) in Von Hippel-Lindau (vHL) Patients Bevacizumab (Avastin) in Unresectable/Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease EYE001 to Treat Retinal Tumors in Patients With Von Hippel-Lindau Syndrome Von Hippel-Lindau Disease Genetic Epidemiology Study Treatment of Von Hippel-Lindau (VHL)-Related Hemangioblastoma With PTK787/ZK 222584 TKI 258 in Von Hippel-Lindau Syndrome (VHL) Study of Sunitinib in Patients With Von Hippel-Lindau (VHL) Disease Contrast-enhanced Ultrasound as a Screening Tool for Kidney Cancer in Patients With Von-Hippel Lindau Genetic Study to Identify Gene Mutations in Participants Previously Enrolled in Clinical Trial NCI-99-C-0053 Who Have Von Hippel-Lindau Syndrome or Are at Risk for Von Hippel-Lindau Syndrome PT2385 for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma Natural History and Management of Von Hippel-Lindau (VHL) Associated Pancreatic Neuroendocrine Tumors Visualizing Vascular Endothelial Growth Factor (VEGF) Producing Lesions in Von Hippel-Lindau Disease Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau Syndrome

Brief Title

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)

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).

Study Phase

Phase 2

Study Type


Primary Outcome

Evaluate efficacy

Secondary Outcome

 Evaluate changes in dynamic contrast enhanced magnetic resonance imaging


Von Hippel-Lindau Disease


PTK787/ZK 222584

Study Arms / Comparison Groups

 PTK787/ZK 222584


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

February 2003

Primary Completion Date

June 2006

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

          -  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




18 Years - N/A

Accepts Healthy Volunteers



Novartis, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID

CPTK787 0144

Study Sponsor

Novartis Pharmaceuticals

Study Sponsor

Novartis, Study Chair, Novartis

Verification Date

February 2011