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

Von Hippel-Lindau Disease Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00052013
• Other study ID #: CPTK787 0144
• Status: Completed
• Phase: Phase 2
• First received: January 21, 2003
• Last updated: March 2, 2017
• Start date: February 2003

Study information

• Verified date: February 2011
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. primary completion date: June 2006
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• CNS Hemangioblastoma
• Hemangioblastoma
• Retinal Hemangioblastoma
• Von Hippel-Lindau Disease

Intervention

Drug:
• PTK787/ZK 222584

Locations

Country	Name	City	State
United States	Dana Farber Cancer Institute	Boston	Massachusetts
United States	Duke University Medical Center	Durham	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluate efficacy
Secondary	Evaluate changes in dynamic contrast enhanced magnetic resonance imaging
Secondary	To explore the correlation of the pharmacokinetics
Secondary	To assess changes in surrogate markers of angiogenesis

External Links

•   CPTK787 0144 results on the Novartis Clinical Trials Website