NCT00052013

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).

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_2

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 21, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

January 22, 2003

Completed
10 days until next milestone

Study Start

First participant enrolled

February 1, 2003

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2006

Completed
Last Updated

March 6, 2017

Status Verified

February 1, 2011

Enrollment Period

3.3 years

First QC Date

January 21, 2003

Last Update Submit

March 2, 2017

Conditions

Von Hippel-Lindau DiseaseCNS HemangioblastomaRetinal Hemangioblastoma

Keywords

von Hippel-Lindau disease,CNS,hemangioblastoma,retinal

Outcome Measures

Primary Outcomes (1)

  • Evaluate efficacy

Secondary Outcomes (3)

  • Evaluate changes in dynamic contrast enhanced magnetic resonance imaging

  • To explore the correlation of the pharmacokinetics

  • To assess changes in surrogate markers of angiogenesis

Study Arms (1)

PTK787/ZK 222584

EXPERIMENTAL
Drug: PTK787/ZK 222584

Interventions

PTK787/ZK 222584DRUG
PTK787/ZK 222584

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

MeSH Terms

Conditions

von Hippel-Lindau DiseaseHemangioblastoma

Interventions

vatalanib

Condition Hierarchy (Ancestors)

Neurocutaneous SyndromesNervous System DiseasesAngiomatosisVascular DiseasesCardiovascular DiseasesCiliopathiesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, InbornHemangioma, CapillaryHemangiomaNeoplasms, Vascular TissueNeoplasms by Histologic TypeNeoplasms

Study Officials

  • Novartis

    Novartis

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

January 21, 2003

First Posted

January 22, 2003

Study Start

February 1, 2003

Primary Completion

June 1, 2006

Last Updated

March 6, 2017

Record last verified: 2011-02

Locations

US(2)