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PTK787/ZK 222584

Phase 2

Von Hippel-Lindau Disease | Small molecule | Oncology |Novartis AG|Last Updated: Mar 6, 2017

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment11
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00052013Treatment of Von Hippel-Lindau (VHL)-Related Hemangioblastoma With PTK787/ZK 222584PHASE2 COMPLETED 11Feb 1, 2003 -Mar 6, 20172 United States
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Study Endpoints
Primary Endpoints
Evaluate efficacy
Secondary Endpoints
Evaluate changes in dynamic contrast enhanced magnetic resonance imaging
To explore the correlation of the pharmacokinetics
To assess changes in surrogate markers of angiogenesis
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PTK787/ZK 222584EXPERIMENTAL -
Interventions
NameTypeDescription
PTK787/ZK 222584DRUG -
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites2

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 t...

Countries:United States
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