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

Sponsor:	Novartis Pharmaceuticals
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00052013

Purpose

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 Central Nervous System Capillary Hemangioblastoma Retinal Capillary Hemangioblastoma	Drug: PTK787/ZK 222584	Phase II

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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:

Genetics Home Reference related topics: von Hippel-Lindau syndrome

MedlinePlus related topics: Von Hippel-Lindau Disease

Drug Information available for: Vatalanib

U.S. FDA Resources

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

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00052013

Locations

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

Investigators

Study Chair:

Novartis

More Information

No publications provided

Responsible Party:	External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00052013 History of Changes
Other Study ID Numbers:	CPTK787 0144
Study First Received:	January 21, 2003
Last Updated:	February 16, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Novartis:

von Hippel-Lindau disease,
CNS,
hemangioblastoma,
retinal

Additional relevant MeSH terms:

Von Hippel-Lindau Disease
Hemangioblastoma
Neurocutaneous Syndromes
Nervous System Diseases
Angiomatosis
Vascular Diseases
Cardiovascular Diseases
Hemangioma, Capillary
Hemangioma

Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Neoplasms
Vatalanib
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on February 12, 2012