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

Sponsored by:	Novartis
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 CNS Hemangioblastoma Retinal Hemangioblastoma	Drug: PTK787/ZK 222584	Phase II

Genetics Home Reference related topics: von Hippel-Lindau syndrome

MedlinePlus related topics: Von Hippel-Lindau Disease

Drug Information available for: Vatalanib Butanedioic acid, compd. with N-(4-chlorophenyl)-4-(4-pyridinylmethyl)-1-phthalazinamine (1:1)

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
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)

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

Estimated Enrollment:	11
Study Start Date:	January 2003

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

Investigators

Study Chair:

Novartis

More Information

Study ID Numbers:	CPTK787 0144
Study First Received:	January 21, 2003
Last Updated:	September 21, 2006
ClinicalTrials.gov Identifier:	NCT00052013
Health Authority:	United States: Food and Drug Administration

Study placed in the following topic categories:

Angiomatosis
Von Hippel-Lindau Disease
Hemangioblastoma
Hemangioma, Capillary
Von Hippel-Lindau syndrome

Hemangioma
Vascular Diseases
Vatalanib
Neurocutaneous Syndromes

Additional relevant MeSH terms:

Neoplasms
Neoplasms by Histologic Type
Molecular Mechanisms of Pharmacological Action
Nervous System Diseases
Neoplasms, Vascular Tissue

Enzyme Inhibitors
Cardiovascular Diseases
Protein Kinase Inhibitors
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009